Cystic Fibrosis Gene Therapy
A short animation to briefly explain the research being conducted at the Gene Therapy Department of the Adelaide Women #39;s and Children #39;s Hospital. Donations c...
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Category Archives: Gene Therapy
Cancer set to become treatable: expert
Advances in gene therapy and the deepening understanding of cancer will see the oft-fatal disease becoming treatable in two decades, said cancer researcher Inder M. Verma.
Cancer mutations are being exposed cancer is in retreat through a combination of surgery, radiation, chemotherapy, molecular and genetic therapy, cancer will become a chronic disease rather than a terminal one, said Dr. Verma, a professor in Laboratory of Genetics at the Salk Institute for Biological Studies, at the Infosys Science Foundation Lecture at the National Centre for Biological Sciences here on Wednesday.
His optimism was elaborated through an intriguing cat-and-mouse game that played out for over five years of research into the Glioblastomas multiforme (GBM), a lethal form of brain cancer that kills the patient within 14 months.
Understanding GBM was critical as relapse, even after surgery or treatment, was certainty, said Dr. Verma.
The researchers at the Salk Institute developed a novel genetic technique to switch on genes in around five cells of a mouse brain to make them into cancer cells. The cells grew to all parts of the brain, but more importantly, they started to exhibit stem cell characteristics, said Dr. Verma.
Unlike the normal cell, a stem cell can divide into specialised cells a phenomenon that explains the resurgent ability of the GBM cancer. Even if you surgically remove the tumour, one cell is enough to recreate the cancer again, he explained.
Using gene therapy, the team of scientists attempted to block this ability as well as use drugs to block blood supply to the cancer cell. While the tumour did become smaller, it became even more invasive. Though the treatment did not work, the cancer cell did reveal the genes responsible for its invasiveness.
We began to genetically cut out the cancers invasiveness, and for the first time, experiments showed GBM cancer could be controlled This is an exciting area that can be possibly used to treat other forms of cancer, said Dr. Verma.
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Neuropathy: Relief for diabetics with painful condition
Walking barefoot on sand "felt like walking on glass" for Keith Wenckowski, who has lived with type-one diabetes for more than two decades.
One of the participants in a new Northwestern Medicine study who suffered from painful diabetic neuropathy (PDN), Wenckowski finally found relief from the constant foot pain that required him to wear shoes at all times, even to the beach.
The study found that those with PDN who received two low dose rounds of a non-viral gene therapy called VM202 had significant improvement of their pain that lasted for months.
"I can now go to a beach and walk on the sand without feeling like I am walking on glass," Wenckowski said.
The results of this phase two, double-blind, placebo-controlled study will be published March 5 in the journal Annals of Clinical and Translation Neurology.
Right now there is no treatment for this disease of the peripheral nerves that affects 20 to 25 percent of diabetics. Patients with the most extreme form of the disease feel intense pain with a slight graze or touch. The pain can interfere with daily activities, sleep, mood and can diminish quality of life.
"Those who received the therapy reported more than a 50 percent reduction in their symptoms and virtually no side effects," said Dr. Jack Kessler, lead author of the study. "Not only did it improve their pain, it also improved their ability to perceive a very, very light touch."
Kessler is the Ken and Ruth Davee Professor of Stem Cell Biology in the department of neurology and a professor in the department of pharmacology at Northwestern University Feinberg School of Medicine. He also is an attending physician at Northwestern Memorial Hospital.
VM202 contains human hepatocyte growth factor (HGF) gene. Growth factor is a naturally occurring protein in the body that acts on cells -- in this case nerve cells -- to keep them alive, healthy and functioning. Future study is needed to investigate if the therapy can actually regenerate damaged nerves, reversing the neuropathy.
Wenckowski had continuous numbness, but now, more than a year since he received the therapy, his symptoms have not returned. "I am hoping the effects I am feeling do not cease," he said.
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Gene Therapy Market Report 2014-2024 – Technologies, Markets and Companies
DUBLIN, Mar. 03, 2015 /PRNewswire/ --Research and Markets
(http://www.researchandmarkets.com/research/gxqhg9/gene_therapy) has announced the addition of Jain PharmaBiotech's new report "Gene Therapy - Technologies, Markets and Companies" to their offering.
Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.
Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.
Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2014, over 2050 clinical trials have been completed, are ongoing or have been approved worldwide.A breakdown of these trials is shown according to the geographical areas and applications.
Since the death of Jesse Gelsinger in the US following a gene therapy treatment, the FDA has further tightened the regulatory control on gene therapy. A further setback was the reports of leukemia following use of retroviral vectors in successful gene therapy for adenosine deaminase deficiency. Several clinical trials were put on hold and many have resumed now. The report also discusses the adverse effects of various vectors, safety regulations and ethical aspects of gene therapy including germline gene therapy.
The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2014-2024. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright.The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets
The voluminous literature on gene therapy was reviewed and selected 750 references are appended in the bibliography.The references are constantly updated. The text is supplemented with 75 tables and 15 figures.
Profiles of 181 companies involved in developing gene therapy are presented along with 223 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade.
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Gene Therapy Market Report 2014-2024 - Technologies, Markets and Companies
Global Gene Therapy Market Report 2015-2025 – Extensive Study on the Marketed and Pipeline Gene Therapies
DUBLIN, Mar. 03, 2015 /PRNewswire/ --Research and Markets
(http://www.researchandmarkets.com/research/rcv4lq/gene_therapy) has announced the addition of the "Gene Therapy Market, 2015 - 2025" report to their offering.
The "Gene Therapy Market, 2015-2025" report provides an extensive study on the marketed and pipeline gene therapies. A lot of research has been carried out in this field for over a decade but there are only five approved therapies (four available in Asian markets; one approved in the EU). There are many promising therapies which are currently being developed worldwide; the approach is likely to result in several commercial success stories in the foreseen future. The report covers various aspects, such as key players, marketed gene therapy products, products in clinical / pre-clinical research, associated ethical issues, likely future developments and upcoming opportunities for a variety of stakeholders.
Several disorders that arise inside the body are a result of either a direct genetic aberration or a dysfunctional/non-functional protein. The attempt to use nucleic acids to correct or delete the genes causing a particular disease is known as gene therapy. Although gene therapy has not contributed significantly to the global pharmaceutical market yet, it is anticipated to grow at a fast pace over the next decade.
Gendicine, developed by SiBiono GeneTech, was the foremost gene therapy that entered market in 2004 in China. Since then four more therapies have received approval in China, Philippines, Russia and the EU. This number for approved / marketed therapies seems weak at present; however, the strong and highly populated pipeline holds tremendous potential. There are 12 gene therapies in late stage of clinical development for the treatment of cancer, ocular and cardiovascular disorders.
There are several concerns that remain to be answered; examples include insertional mutagenesis, treatment of multigene disorders, curbing the risk of immune reactions, eugenics, high cost of therapy and ethical concerns related to making alterations at the genetic level. Despite this, gene therapy does offer a ray of hope for patients who either have no treatment options or show no benefits with drugs that are currently available. Such a benefit far outweighs any disadvantages that may be associated with this upcoming therapeutic field.
Key Topics Covered:
1. Preface
2. Executive Summary
3. Introduction
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New Gene Therapy Procedure Could Hold Key to Preventing Blindness for Certain Patients
Carlsbad, CA (PRWEB) February 28, 2015
A study out of the University of Oxford has developed a promising new gene therapy procedure for the treatment of a hereditary ocular condition that leads to progressive blindness in patients. Authored by Dr. Robert MacLaren, professor of ophthalmology at the University of Oxford and published in the Lancet Medical Journal on January 16, 2014, the study treated six patients suffering from choroideremia, a rare genetic disorder that mostly affects men and leads to the degeneration of the choroid and retina, eventually causing blindness. Night blindness and loss of peripheral vision are the most common symptoms of choroideremia. For the study, researchers injected the subjects with healthy genetic material in an attempt to repair the damaged portions of the patients own gene, halting the onset of blindness.
Developing technologies and gene therapy are the future of ophthalmology, said Carlsbad LASIK surgeon Dr. Michael Tracy. In the past, there was little that could be done for certain patients facing blindness due to genetic and degenerative conditions. Gene therapy can lead to treatments that can stop the onset of blindness in certain patients before its too late.
For the Oxford study, researchers used virus particles to add the genetic material to the fluid behind the retina. The goal of the therapy was for the new genetic material to fix or patch flaws in the patients damaged DNA. Timing was an important element of the study. The participants in the study were all treated before their visual acuity had been irreversibly diminished, giving the therapy a greater chance of success. Of the six participants, two reported significant improvement in visual acuity, the other four reported improvement in night vision and maintained the same level of visual acuity as before the treatment in the affected eye. The therapy does involve the risk of further or permanent damage as it requires detachment of the retina, but none of the participants reported further degeneration of visual acuity in the treated eye.
This research is very important because it can pave the way for more gene therapy studies for treating other debilitating conditions that can lead to blindness, such as macular degeneration, added Dr. Tracy.
Dr. Michael Tracy is a board-certified ophthalmologist in Carlsbad, CA. He completed his residency training in ophthalmology at the Scheie Eye Institute at the University of Pennsylvania and his fellowship in Corneal and Refractive Surgery at the Bascom Palmer Eye Institute. Dr. Tracy specializes in LASIK and PRK surgery at his private practice at Carlsbad Eye Care.
To contact Carlsbad Eye Care, please visit http://carlsbad-eye-care.com/ or call (760) 603-9910.
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New Gene Therapy Procedure Could Hold Key to Preventing Blindness for Certain Patients
Pancreatic cancer patients who benefit from personalized treatment identified
Cancer researchers at Indiana University report that about 15 percent of people with pancreatic cancer may benefit from therapy targeting a newly identified gene signature.
Using data from the Cancer Genome Atlas, Murray Korc, M.D., the Myles Brand Professor of Cancer Research at the Indiana University School of Medicine and a researcher at the Indiana University Melvin and Bren Simon Cancer Center, and colleagues found that a sub-group of pancreatic cancer patients who possess a strong angiogenic gene signature could benefit from personalized therapies that cut off the pathways that feed the cancer's growth.
This particular gene signature enables abnormal blood vessels to form in tumors, which feeds the tumor's growth.
The finding, published online Feb. 25 in the journal Oncotarget, is new because the prevalence of this signature was not previously known. The authors also demonstrated for the first time that endothelial cells, the main type of cell found in the inside lining of blood vessels, can produce molecules that directly stimulate the growth of pancreatic cancer cells.
"We showed that endothelial cells can stimulate the growth of pancreatic cancer cells and that by silencing or inhibiting certain pathways -- JAK1-2 and STAT3 -- we can alter that effect," Dr. Korc explained. "We demonstrated that it is possible to target these pathways and prolong the survival of genetically modified mice whose pancreatic cancers also have a strong pro-angiogenic gene signature."
Thus, for people with a strong pro-angiogenic gene signature, the finding suggests that they may benefit from targeted therapy that is directed against one of these pathways.
An important feature of the study was to demonstrate that it is possible to implant in mice small biopsy samples obtained from patients undergoing endoscopic procedures and to generate human tumors in these mice. When the original human tumor had evidence for angiogenesis, the implanted human tumor also exhibited angiogenesis in the mouse. Additional studies are necessary to confirm that these approaches could guide the design of precision medicine using targeted therapies, Dr. Korc said.
The need for new therapies for pancreatic cancer patients is great as only 7 percent of people with the disease survive more than five years after diagnosis. According to the American Cancer Society, there will be an estimated 48,960 new cases of pancreatic cancer and 40,560 deaths from the disease in 2015.
Co-authors of the study were Jesse Gore, Ph.D.; Stuart Sherman, M.D.; Harvey Cramer, M.D.; Hai Nguyen, M.D.; Kelly Craven, Monica Cheng, and Julie Wilson, all of IU School of Medicine, and Gregory Cote M.D. M.S., formerly of IU School of Medicine and now at the Medical University of South Carolina.
The study was made possible, in part, by grant CA-075059 awarded by the National Cancer Institute of the National Institutes of Health.
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Pancreatic cancer patients who benefit from personalized treatment identified
'Gene Therapy Cardiovascular Insight: Pipeline Assessment, Market Trend, Technology and Competitive Landscape'
Albany, NewYork (PRWEB) February 25, 2015
Researchmoz.us announces the latest addition to its growing database of analytical market research reports. The new report, titled Gene Therapy Cardiovascular Insight: Pipeline Assessment, Market Trend, Technology and Competitive Landscape, looks into the value chain of gene therapy and its association with cardiovascular treatments.
The report goes over the fundamentals of gene therapy in brief, explaining its biological mechanism and providing some historic background to better understand the growing influence of gene therapy in the healthcare sector and more particularly in the application of cardiovascular gene therapy. It also covers some of the jargon of the gene therapy sector for better comprehension of the detailed discussion of pipeline products.
Read Full Report With TOC at http://www.researchmoz.us/gene-therapy-cardiovascular-insight-pipeline-assessment-market-trend-technology-and-competitive-landscape-report.html
The report goes on to describe the determinant conditions in the cardiovascular gene therapy market, including the competitive landscape of the market and a detailed examination of the potential impact of market drivers and restraining factors. It describes the major products currently dominating the cardiovascular gene therapy market as well as the most potentially influential products and therapies in the pipeline. The analysis of the major pipeline products includes an evaluation of the competitive landscape concerning the products in questionthe market players developing them and the impact they could have on the markets competitive hierarchy.
The report elaborates upon the product description and technological background of the cardiovascular gene therapy in question, as well as the corporate aspect of it such as development partners and licensors and collaborators, and the stage of development which the product currently occupies.
The publication also includes a detailed overview of the pre-clinical and clinical outcomes of gene therapies and various biochemical aspects of the therapy such as the vector used in the procedure, the gene targeted by the therapy and localization, and an in-depth explanation of the mechanism by which the therapy operates. The report also covers dormant and discontinued products, which helps gain an understanding of what the market isnt ready for, or wasnt ready for at the time of the discontinuation. The pipeline cardiovascular gene therapies covered in the report include Gendicine, Rexin G, and Glybera.
Through the detailed analysis of all aspects related to the cardiovascular gene therapy market, the report provides in-depth insights on which to base winning market strategies.
In all, the report covers more than 25 cardiovascular gene therapy products from more than 20 manufacturers. The companies use 8 distinct technological support systems that have their own set of strengths and weaknesses, which are profiled in the report. As far as vectors are concerned, 50% of the total vectors studies are non-viral vectors, 46% are viral vectors, while a minuscule 4% are RNAi therapeutics.
All Latest Market Research Report at http://www.researchmoz.us/latest-report.html
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A Stem Cell Cure for Bubble Baby Disease (SCID) – Video
A Stem Cell Cure for Bubble Baby Disease (SCID)
Visit: http://www.uctv.tv/) On November 18th, 2014, a UCLA research team led by Donald Kohn, M.D., announced a breakthrough gene therapy and stem cell cure ...
By: University of California Television (UCTV)
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Cancer Gene Therapy — 8News Anchor Amy Lacey/VCU Massey Cancer Center – Video
Cancer Gene Therapy -- 8News Anchor Amy Lacey/VCU Massey Cancer Center
It can be devastating to be diagnosed with cancer, but what if you could take drugs tailored to your DNA, drugs that can buy you precious time? 8News Anchor Amy Lacey has more on the new test...
By: Amy Lacey
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Cancer Gene Therapy -- 8News Anchor Amy Lacey/VCU Massey Cancer Center - Video
Gene therapy leads to promising avenue for HIV vaccine – Video
Gene therapy leads to promising avenue for HIV vaccine
Gene therapy leads to promising avenue for HIV vaccine.
By: 7BILLIONHUMANBEING
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Gene therapy leads to promising avenue for HIV vaccine - Video
Alliance for Cancer Gene Therapy (ACGT) Thanks Dr. Savio L.C. Woo For His Service; Names Dr. Joseph Glorioso …
Stamford, CT (PRWEB) February 20, 2015
World-renowned scientist and University of Pittsburgh School of Medicine genetics and biochemistry professor Joseph Glorioso III, PhD has been named Chairman of the Scientific Advisory Council at Alliance for Cancer Gene Therapy.
Glorioso, known for his work on the molecular and genetic aspects of the herpes simplex virus and how to better engineer this organism as a vector for transporting therapeutic genes, will take the helm from Dr. Savio L.C. Woo, the founding Chairman of ACGTs Scientific Advisory Council; Dr. Woo has also been named Chairman Emeritus of the Council.
Savio has been a remarkable and dynamic leader who has steered the Council from the beginning to focus on young investigators and groundbreaking clinical translation, Glorioso said. This work has resulted in ACGT pioneering breakthroughs in gene and cell therapy treatments for cancer. We now look forward, to expanding our vision to include later-stage research, which will be very exciting.
Glorioso received his bachelors degree and doctorate from Louisiana State University before joining the University of Michigan Medical School in the late 1970s. He attained the rank of professor and assistant dean for research and graduate studies there, and then joined the University of Pittsburgh School of Medicine in 1989. At Pittsburgh, Glorioso served as Professor and Chair of the Department of Molecular Genetics and Biochemistry, as well as the McEllroy Professorship in Biochemistry until 2009. He continues his groundbreaking work in the development of herpes viral vectors for the treatment of cancer, chronic pain and diseases of the central nervous system. Glorioso has served on ACGTs Scientific Advisory Council since 2005.
Joe has been a valuable member of our Scientific Advisory Council and has made tremendous strides in his own work to improve the quality of life for patients fighting cancer and other diseases; that same vision will advance ACGTs own pursuit of effective cell and gene therapy treatments, said Barbara Netter, President and Co-founder of ACGT.
ACGTs Scientific Advisory Council, composed of preeminent physicians and researchers in cell and gene therapy, serves without remuneration and establishes the scientific criteria for the review of all grants. Council members are also tasked with developing strict accountability guidelines requiring periodic progress reports. At present, the Council is composed of 15 members, including Glorioso.
Based in Stamford, Connecticut, ACGT funds top physicians and researchers at medical institutions in the U.S. and Canada. The Foundation supports a number of gene and cell therapy treatments, including immunotherapy, which activates patients own immune systems to battle cancerous cells. In 2014, the FDA granted fast-track status to 2 immunotherapy treatments for leukemia, for which ACGT provided critical early funding.
About Alliance for Cancer Gene Therapy (ACGT): Established in 2001, ACGT (http://www.acgtfoundation.org) is the nations only not-for-profit dedicated exclusively to cell and gene therapy treatments for all types of cancer. One-hundred percent of contributions go directly to research. ACGT has funded 46 grants in the U.S. and Canada since its founding in 2001 by Barbara Netter, President, and her late husband, Edward, to conduct and accelerate critically needed innovative research. Since its inception, ACGT has awarded 31 grants to Young Investigators and 15 grants to Clinical Investigators, totaling more than $25 million in funding. ACGT is located at 96 Cummings Point Road, Stamford, CT 06902.
ACGT on Facebook: http://www.facebook.com/ACGTfoundation ACGT on Twitter: http://www.twitter.com/ACGTfoundation ACGT on YouTube: http://www.youtube.com/user/ACGTfoundation
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Uptown Funk Gene Therapy Parody – Video
Uptown Funk Gene Therapy Parody
This is a video made for a 9th grade biology "celebration" grade Have fun???
By: Andrew Melton
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The Clinical Promise of the Gene Therapy from Harrison’s Principles of IM, 19th Edition – Video
The Clinical Promise of the Gene Therapy from Harrison #39;s Principles of IM, 19th Edition
Dr. J. Larry Jameson, editor for Harrison #39;s Principles of Internal Medicine, discusses the clinical promise of the gene therapy. Visit http://www.HarrisonsIM.com to watch more videos of Dr. J. Larry...
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The Clinical Promise of the Gene Therapy from Harrison's Principles of IM, 19th Edition - Video
Uptown Funk Gene Therapy – Video
Uptown Funk Gene Therapy
This video is about Uptown Funk Gene Therapy.
By: Andrew Melton
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Gene study finds link with implications for melanoma therapy
Lawrence, KS (PRWEB) February 18, 2015
Pediatric and Developmental Pathology An odd-looking or changing mole or birthmark is always worrisome, particularly on a child. While most are benign, cancer jumps into every parents mind. Gene studies and potential related therapies are just one front in the wide-ranging battle against melanoma.
An analysis in the new issue of the journal Pediatric and Developmental Pathology looks at the link between the typical clinical features of two diseases and the status of two genes. In their study, researchers confirmed that a mutation of the BRAF gene affects people with large or giant birthmarks known as congenital melanocytic nevi (CMNs) and, for the first time, they also found this mutation present in neurocutaneous melanocytosis, a rare neurological disorder associated with Large/Giant CMNs. They suggest that it may be possible to target the BRAF gene during therapy.
CMNs are literally birthmarks, developing in the womb as a fetus grows. Such a dark-colored, sometimes hairy, patch of skin can continue to develop as the child grows, and if it reaches 20 cm or more, it is classified as large or giant. Although usually benign at birth, they can associate with a range of other problems, from decreased sweating and tissue growth to a greater likelihood of melanoma, a skin cancer that is becoming increasingly common worldwide among adults and even teenagers. Neurocutaneous melanocytosis, in which the pigment cells rapidly spread across the brain, spinal cord and meninges, can also develop.
The current study collected pigmented tissue from 66 patients with Large/Giant CMN. The authors first looked for the more commonly mutated NRAS gene and then evaluated those tissues without NRAS mutation for BRAF mutation.
The authors found a new link between BRAF mutations and aggressive disease pattern. They learned of a definite association between the BRAF gene and large or giant CMNs. They also discovered, for the first time, that some patients with neurocutaneous melanocytosis had BRAF but not NRAS mutation. Race was a factor in the appearance of a mutational group, with Asians less likely to show the BRAF mutation.
Surprisingly, more nodules were found in the pigmented skin among people who had the BRAF mutation than among those with the NRAS mutation. Less hair was also found with the BRAF mutation.
The authors concluded that not only NRAS mutations can occur in the studied conditions. They also learned that BRAF mutations may be responsible for more serious disease than previously thought. They said that this means it may be possible to use BRAF inhibitors to target the mutated BRAF gene when treating patients with large or giant CMNs.
Full text of the article BRAF mutations are also associated with neurocutaneous melanocytosis and large/giant congenital melanocytic nevi, Pediatric and Developmental Pathology, Vol. 18, No. 1, 2014, is now available.
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Gene study finds link with implications for melanoma therapy
Shift 12 – Episode 34: To fight or not – Video
Shift 12 - Episode 34: To fight or not
There is no single treatment for cancer, and patients often receive a combination of therapies and palliative care. Treatments usually fall into one of the following categories: surgery, radiation,...
By: SABC Education Shows
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UD alumni $2.5 million closer to saving 5-year-old Eliza – Video
UD alumni $2.5 million closer to saving 5-year-old Eliza
In 2014, University of Delaware alumni Glenn and Cara O #39;Neill raised half of the $5 million needed for a gene therapy clinical trial and the medicine necessa...
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UD alumni $2.5 million closer to saving 5-year-old Eliza - Video
Synthetic biology yields new approach to gene therapy
10 hours ago by Amanda Siegfried Dr. Leonidas Bleris (left), assistant professor of bioengineering at UT Dallas,and Richard Taplin Moore MS11 helped create a new delivery system that may change gene therapy.
Bioengineers at The University of Texas at Dallas have created a novel gene-delivery system that shuttles a gene into a cell, but only for a temporary stay, providing a potential new gene-therapy strategy for treating disease.
The approach offers distinct advantages over other types of gene therapies under investigation, said Richard Taplin Moore MS'11, a doctoral student in bioengineering in the Erik Jonsson School of Engineering and Computer Science. He is lead author of a study describing the new technique in the Jan. 30 issue of the journal Nucleic Acids Research.
"In other gene therapy approaches, the therapeutic genetic messages being delivered can persist for a long time in the patient, potentially lasting for the patient's entire lifetime," Moore said. "This irreversibility is one reason gene therapies are so difficult to get approved."
The UT Dallas study describes proof-of-concept experiments in which a gene carrying instructions for making a particular protein is ordered to self-destruct once the cell has "read" the instructions and made a certain quantity of the protein. In its experiments with isolated human kidney cells, the research team successfully deliveredand then destroyeda test gene that makes a red fluorescent protein.
More research is needed to determine whether and how well the system might work in living organisms. But Moore said the ultimate goal is to refine the method to deliver genes that produce therapeutic proteins or drugs. The nature of the gene delivery system offers more control over how much protein the gene produces in cells or tissues. Because it does not alter the cell permanently, the method also sidesteps potential health problems that can occur if a gene is delivered to the wrong place in a cell's genome.
"Our goal was to create a delivery system for therapeutic genes that would self-destruct, giving us more control over the delivered DNA by limiting the time it resides in cells," Moore said.
Located in the nucleus of each human cell, genes are made of DNA and contain instructions for making proteins. Machinery inside each cell "reads" the instructions and builds those proteins, which then carry out various functions needed to sustain life. Defective or mutated genes can result in malfunctioning or missing proteins, leading to disease.
Gene therapy aims to replace defective genes with healthy versions. Typically the good genes are packaged with a delivery mechanism called a vector, which transports the genetic material inside cells. With traditional approaches, once in the cell, the gene permanently integrates itself into the cell's DNA.
Although promising, this type of gene therapy also has risks. If a therapeutic gene is inserted in the wrong place in the cell's DNA, such as too close to a cancer-related gene, the process could activate additional disease-causing genes, resulting in lifelong health problems for the patient. While many gene therapy clinical trials are underway worldwide, the Food and Drug Administration has not approved for sale any human gene therapy product in the U.S.
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Cancer gene therapy advances
Flow chart of one method of engineering T cells to fight cancer.
Tough blood cancers are responding to treatment with the patient's own genetically engineered immune cells, according to a cancer specialist who is helping test the bold -- and risky -- approach.
The treatment has produced complete remissions in large percentages of patients treated, up to 90 percent in one group of 30 patients. Moreover, these are all extremely sick patients, whose cancer has resisted other therapies, leaving them with virtually no options. The longest survivor has been in complete remission for more than 4 years, said Dr. David Porter of the University of Pennsylvania.
Porter spoke Sunday at the 35th Annual Conference on Clinical Hematology & Oncology, held in La Jolla by Scripps Health. Speakers like Porter came from around the country to discuss advances in their field, part of a continuing medical education program to keep doctors up to date with the latest medical advances. The conference continues through Tuesday.
Dr. David Porter / University of Pennsylvania
Porter works with Dr. Carl June and other colleagues to hone the effectiveness of using T cells genetically programmed to attack malignant B cells. B cell malignancies cause such cancers as chronic lymphocytic leukemia, or CLL; acute lymphoblastic leukemia, or ALL, and non-Hodgkin's lymphoma.
The genetically altered T cells are given an artificially created gene to produce a chimeric antigen receptor. This receptor recognizes the protein CD19, produced almost exclusively on B cells. The T cells hone in on this protein and kill the B cells. When the cancer is knocked down, some of the T cells remain behind, ready to pounce on any recurrence.
The trials are now expanding to include myloma, Porter said. For more information on the university's T cell-based clinical trials, go to http://www.penncancer.org/tcelltherapy.
June's team began testing the therapy in 2010 in CLL patients and then in those with ALL. These were all patients with relapsed cancers that had become resistant to other therapies. The therapy has been progressively refined, Porter said. Other centers have developed their own versions of this therapy.
In October, 2014, the University of Pennsylvania and Children's Hospital of Philadelphia announced that 27 out of 30 patients with relapsed ALL achieved a complete remission within one month of treatment, and 23 were still alive 6 months after treatment. As of October, 19 of the patients remained in remission. Most treated, 25 out of 30, were children. The response rate with CLL is about 47 percent, Porter said.
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