Category Archives: Gene Medicine

3 hours ago RGEN-induced digenome sequencing to capture off-target sites. (a) Overview of Digenome-seq. Forward and reverse sequence reads are shown in pink and blue, respectively. Red triangles and vertical lines indicate cleavage positions. WT, wild type. (b) Representative IGV images obtained using the HBB-specific RGEN at the on-target site.

The IBS research team (Center for Genome Engineering) has successfully confirmed that CRISPR-Cas9 has accurate on-target effects in human cells, through joint research with the Seoul National University College of Medicine and ToolGen, Inc.

There has been great interest in CRISPR-Cas9 as a tool to develop anticancer cell therapies or to correct genetic defects that cause hereditary in stem and somatic cells. However, since there has been no reliable and sensitive method to measure the accuracy of CRISPR-Cas9 genome-wide, its safety has remained in question. Consequently, it has been difficult to eliminate the possibility that CRISPR-Cas9 may induce mutations in off-target sequences that are similar to on-target sequences. Off-target mutations in tumor suppressor genes, for example, can cause cancer.

The researchers have developed a technique termed Digenome-seq to locate both on-target and off-target sequences that can be mutated by CRISPR-Cas9 via genome sequencing. They digested human genomic DNA using Cas9 nucleases in a test tube, which was then subjected by whole genome sequencing. This in vitro digest yielded a unique pattern at both on-target and off-target sequences that can be computationally identified. Furthermore, by adding guanine nucleotides at the end of sgRNA(single guided RNA) that composes CRISPR-Cas9, they have successfully created this highly-developed programmable nuclease, which has no measurable off-target effects in the human genome.

Jin-Soo Kim, the director of the Center for Genome Engineering at IBS, as well as the professor of the Department of Chemistry at Seoul National University says, "If CRISPR-Cas9 truncates off-target DNA sequences, it might induce unwanted mutations. Since we have succeeded in confirming the accuracy of CRISPR-Cas9, we anticipate that there will be a great progress in the development of gene or cell therapies," emphasizing the significance of this research achievement.

Nature Methods has also highlighted this achievement as one of the "2015 Methods to Watch" in its January issue.

Explore further: Revolutionizing genome engineering: Review on history and future of the CRISPR-Cas9 system published

More information: Daesik Kim, Sangsu Bae, Jeongbin Park, Eunji Kim, Seokjoong Kim, Hye Ryeong Yu, Jinha Hwang, Jong-Il Kim & Jin-Soo Kim.(2015) Nature Methods. DOI: 10.1038/nmeth.3284

Journal reference: Nature Methods

Provided by Institute for Basic Science

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End of CRISPR-CAS9 controversy

A gene is the molecular unit of heredity of a living organism. It is used extensively by the scientific community as a name given to some stretches of deoxyribonucleic acids (DNA) and ribonucleic acids (RNA) that code for a polypeptide or for an RNA chain that has a function in the organism. Living beings depend on genes, as they specify all proteins and functional RNA chains. Genes hold the information to build and maintain an organism's cells and pass genetic traits to offspring. All organisms have genes corresponding to various biological traits, some of which are instantly visible, such as eye color or number of limbs, and some of which are not, such as blood type, increased risk for specific diseases, or the thousands of basic biochemical processes that comprise life. The word gene is derived from the Greek word genesis meaning "birth", or genos meaning "origin" (see pangenesis).

A modern working definition of a gene is "a locatable region of genomic sequence, corresponding to a unit of inheritance, which is associated with regulatory regions, transcribed regions, and or other functional sequence regions ".[1][2] Colloquial usage of the term gene (e.g., "good genes", "hair color gene") may actually refer to an allele: a gene is the basic instruction a sequence of nucleic acids (DNA or, in the case of certain viruses RNA), while an allele is one variant of that gene. Thus, when the mainstream press refers to "having" a "gene" for a specific trait, this is customarily inaccurate. In most cases, all people would have a gene for the trait in question, although certain people will have a specific allele of that gene, which results in the trait variant. Further, genes code for proteins, which might result in identifiable traits, but it is the gene (genotype), not the trait (phenotype), which is inherited.

Big genes are a class of genes whose nuclear transcript spans 500 kb (1 kb = 1,000 base pairs) or more of chromosomal DNA. The largest of the big genes is the gene for dystrophin, which spans 2.3 Mb. Many big genes have modestly sized mRNAs; the exons encoding these RNAs typically encompass about 1% of the total chromosomal gene region in which they occur.

The existence of genes was first implied from the work of Gregor Mendel (18221884), who, between the years of 1857 to 1864 planted 8000 common edible pea plants and studied and tabulated the inheritance patterns in peaplants (Pisum) tracking inheritance of traits from parent to offspring and describing these mathematically as 2n combinations where n is the number of differing characteristics in the original peas. Although he did not use the term gene, he explained his results in terms of inherited characteristics. The notion of a gene[3] is evolving with the science of genetics, but began when Mendel noticed that biological variations are inherited from parent or grandparent organisms as specific, discrete traits and are transmitted thus unaltered from the original source. Prior to Mendel's work, the dominant theory of heredity was one of blending inheritance, pangenesis, which suggested that each parent contributed fluids to the fertilisation process and that in meiosis the traits of the parents blended and mixed to produce the offspring. Although Mendel's work was largely unrecognized after its first publication in 1866, it was 'rediscovered' in 1900 by three European scientists, Hugo de Vries, Carl Correns, and Erich von Tschermak, who claimed to have reached similar conclusions in their own research. However, these scientists were not yet aware of the identity of the 'discrete units' on which genetic material resides. The biological entity responsible for defining traits was later termed a gene, but the biological basis for inheritance remained unknown until DNA was identified as the genetic material in the 1940s. Mendel was also the first to show independent assortment, the distinction between dominant and recessive traits, the distinction between a heterozygote and homozygote, the phenomenon of discontinuing inheritance and what would later be described as genotype (the genetic material of an organism) and phenotype (the visible traits of that organism) and the conversion of one form into another within few generations.

Charles Darwin used the term gemmule to describe a microscopic unit of inheritance, and what would later become known as chromosomes had been observed separating out during cell division by Wilhelm Hofmeister as early as 1848. The idea that chromosomes are the carriers of inheritance was expressed in 1883 by Wilhelm Roux. Darwin also coined the word pangenesis by (1868).[4] The word pangenesis is made from the Greek words pan (a prefix meaning "whole", "encompassing") and genesis ("birth") or genos ("origin").

Mendel's concept was given a name by Hugo de Vries in 1889, in his book Intracellular Pangenesis; although probably unaware of Mendel's work at the time, he coined the term "pangen" for "the smallest particle [representing] one hereditary characteristic".[5]Danish botanist Wilhelm Johannsen coined the word "gene" ("gen" in Danish and German) in 1909 to describe the fundamental physical and functional units of heredity,[6] while the related word genetics was first used by William Bateson in 1905.[7] He derived the word from de Vries' "pangen". In the early 1900s, Mendel's work received renewed attention from scientists. In 1910, Thomas Hunt Morgan showed that genes reside on specific chromosomes. He later showed that genes occupy specific locations on the chromosome. With this knowledge, Morgan and his students began the first chromosomal map of the fruit fly Drosophila. In 1928, Frederick Griffith showed that genes could be transferred. In what is now known as Griffith's experiment, injections into a mouse of a deadly strain of bacteria that had been heat-killed transferred genetic information to a safe strain of the same bacteria, killing the mouse.

A series of subsequent discoveries led to the realization decades later that chromosomes within cells are the carriers of genetic material, and that they are made of DNA (deoxyribonucleic acid), a polymeric molecule found in all cells on which the 'discrete units' of Mendelian inheritance are encoded. In 1941, George Wells Beadle and Edward Lawrie Tatum showed that mutations in genes caused errors in specific steps in metabolic pathways. This showed that specific genes code for specific proteins, leading to the "one gene, one enzyme" hypothesis.[7]Oswald Avery, Colin Munro MacLeod, and Maclyn McCarty showed in 1944 that DNA holds the gene's information.[8] In 1952, Rosalind Franklin and Raymond Gosling produced a strikingly clear x-ray diffraction pattern indicating a helical form, and in 1953, James D. Watson and Francis Crick demonstrated the molecular structure of DNA. Together, these discoveries established the central dogma of molecular biology, which states that proteins are translated from RNA which is transcribed from DNA. This dogma has since been shown to have exceptions, such as reverse transcription in retroviruses.

In 1972, Walter Fiers and his team at the Laboratory of Molecular Biology of the University of Ghent (Ghent, Belgium) were the first to determine the sequence of a gene: the gene for Bacteriophage MS2 coat protein.[9]Richard J. Roberts and Phillip Sharp discovered in 1977 that genes can be split into segments. This led to the idea that one gene can make several proteins. Recently (as of 20032006), biological results let the notion of gene appear more slippery. In particular, genes do not seem to sit side by side on DNA like discrete beads. Instead, regions of the DNA producing distinct proteins may overlap, so that the idea emerges that "genes are one long continuum".[1] It was first hypothesized in 1986 by Walter Gilbert that neither DNA nor protein would be required in such a primitive system as that of a very early stage of the earth if RNA could perform as simply a catalyst and genetic information storage processor.

The modern study of genetics at the level of DNA is known as molecular genetics and the synthesis of molecular genetics with traditional Darwinian evolution is known as the modern evolutionary synthesis.

According to the theory of Mendelian inheritance, variations in phenotypethe observable physical and behavioral characteristics of an organismare due in part to variations in genotype, or the organism's particular set of genes, each of which specifies a particular trait. Different forms of a gene, which may give rise to different phenotypes, are known as alleles. Organisms such as the pea plants Mendel worked on, along with many plants and animals, have two alleles for each trait, one inherited from each parent. Alleles may be dominant or recessive; dominant alleles give rise to their corresponding phenotypes when paired with any other allele for the same trait, whereas recessive alleles give rise to their corresponding phenotype only when paired with another copy of the same allele. For example, if the allele specifying tall stems in pea plants is dominant over the allele specifying short stems, then pea plants that inherit one tall allele from one parent and one short allele from the other parent will also have tall stems. Mendel's work demonstrated that alleles assort independently in the production of gametes, or germ cells, ensuring variation in the next generation.

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Gene - Wikipedia, the free encyclopedia

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What is a genetic disease?

A genetic disease is any disease that is caused by an abnormality in an individual's genome. The abnormality can range from minuscule to major -- from a discrete mutation in a single base in the DNA of a single gene to a gross chromosome abnormality involving the addition or subtraction of an entire chromosome or set of chromosomes. Some genetic disorders are inherited from the parents, while other genetic diseases are caused by acquired changes or mutations in a preexisting gene or group of genes. Mutations occur either randomly or due to some environmental exposure.

There are a number of different types of genetic inheritance, including the following four modes:

Single gene inheritance, also called Mendelian or monogenetic inheritance. This type of inheritance is caused by changes or mutations that occur in the DNA sequence of a single gene. There are more than 6,000 known single-gene disorders, which occur in about 1 out of every 200 births. These disorders are known as monogenetic disorders (disorders of a single age).

Some examples of monogenetic disorders include:

Single-gene disorders are inherited in recognizable patterns: autosomal dominant, autosomal recessive, and X-linked.

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Genetic Disease - Symptoms Question: What were the symptoms of a genetic disease in you or a relative?

Genetic Disease - Screening Question: Have you been screened for a genetic disease? Please share your story.

Genetic Disease - Personal Experience Question: Is there a genetic disease in your family? Please share your experience.

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Genetic Disease: Get the Definition of These Disorders

Released: 29-Jan-2015 7:00 PM EST Embargo expired: 2-Feb-2015 5:00 PM EST Source Newsroom: Universite de Montreal Contact Information

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Newswise The discovery of the first gene causing familial scoliosis was announced by an international France-Canada research team today. Mystery surrounds the cause of scoliosis, which is a three dimensional deformation of the vertebral column. Many researchers have been attempting to uncover the origins of this disease, particularly from a genetic point of view, explained leading co-author Dr Florina Moldovan of the University of Montreal and the CHU Sainte Justine research hospital. To date, many genes have been suspected of causing scoliosis amongst different populations, but the gene that causes the familial form of the disease remained unknown. Our discovery of this first causative gene is due to the support of the Fondation Yves Cotrel and our international teamwork, in particular with leading co-author Dr. Patrick Edery of CHU de Lyon hospital and Dr. Pierre Drapeau of the CRCHUM.

A variation in the POC5 gene was initially identified by DNA sequencing (exome sequencing) in the samples Dr Patrick Edery collected from a large French family, of whom several members are affected by idiopathic scoliosis. Others variants of the POC5 gene were detected in scoliotic families and in people whose scoliosis had no precedence in their families. The POC5 gene encodes for a centrosomal protein involved in microtubule-organising centres and cellular polarity, explained first author Dr. Shunmoogum (Kessen) Patten, who undertook his post-doctoral work at the CHU Sainte-Justine and CHUM research centres. The pathogenicity of POC5 variants was documented by using the zebrafish, a well-established genetic animal model that has a spine. This model revealed that the over-expression of mutated human POC5 gene led to the rotational deformation of the anterior-posterior axis of the spine in half of the zebrafish embryos. The deformations are similar to the deformations observed in scoliosis patients.

The data suggest that the mutations are dominant, confirming the human genetic analysis. Interestingly, the protein is strongly expressed in the brain, within very precise structures in the midbrain. This leads the research team to believe that there is an association between the brain and idiopathic scoliosis. This is a very heterogenous disease and probably more than one gene is required for disease expression. This discovery has enabled the identification of the first causative gene and represents an important step towards decoding its genetic causes, Dr. Moldovan said. This crucial first step will open the door to future studies that will identify the complementary genes and pathways that play a role in scoliosis in other populations. In particular, a full portrait of genetic events would enable the perfecting of effective preventative methods and strategies for understanding scoliosis, said Dr. Drapeau.

About this study

The researchers published their article entitled Functional variants of POC5 identified in patients with idiopathic scoliosis in The Journal of Clinical Investigation on February 2, 2015. This international collaborative work was performed with the support of the Fondation Yves Cotrel Institut de France, which has supported Dr Moldovans research since 2006. Crucially, during the past 14 years, Dr. Ederys team and colleagues recruited many families with multiple members affected by scoliosis over the generations. Dr Kessen Patten is a post doctoral researcher at the Universit de Montral and is mentored by Dr. Moldovan and Dr. Drapeau of the CHU Sainte-Justine and CHUM research centres, respectively. His research is supported by Fondation CHU Justine, Fondation des toiles, the Network of Applied Medical Genetics (RMGA), Fonds de recherche du Qubec Sant (FRQS) and the Canada Institutes of Health Research (CIHR). Dr. Florina Moldovan is a full professor in the Faculty of Dentistry -Department of Stomatology at the University of Montreal and a researcher at the CHU Sainte-Justine Research Centre. Dr. Pierre Drapeau is a full professor in the at the universitys Faculty of Medicine -Department of Neurosciences and a researcher at the CHUM Research Centre. The University of Montreal is officially known as Universit de Montral.

Links : Dr Moldovan at the University of Montreal - http://www.medent.umontreal.ca/fr/faculte/prof/florina.moldovan/index.htm

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Discovery of a Gene Responsible for Familial Scoliosis

The White House on Friday unveiled a $215 million program to study genes of a million Americans in various stages of sickness and health, with the hope of gaining vast new insight into diseases and how to cure them.

The Precision Medicine Initiative, which President Barack Obama announced in his State of the Union speech, will bring new funding to federal health and science agencies to build data that flows between medical clinics to labs that sequence the human genome and gather other data. The goal is to find more targeted personalized approaches to treatments and cures sometimes called personalized medicine.

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This helps us find new cures but also helps us create a genuine health care system as opposed to a disease care system. We want each of us to have sufficient information about our particular quirks so that we can make better life decisions, Obama said.

The initiative includes $200 million in new spending for the National Institutes of Health $70 million targeted for the National Cancer Institute and $10 million for the FDA. Another $5 million goes to the federal office in charge of health IT, which will work on making sure the data can be transmitted and shared by different clinical and research centers while protecting patient privacy. The people who allow their DNA to be studied will be volunteers.

Some key Republicans in Congress have signaled support of the precision initiative. House Energy and Commerce Chair Fred Upton (R-Mich.), who this week released draft legislation to reform FDA, said Obamas initiative is a natural fit in the discussion about how to accelerate and improve the discovery, development and delivery of new cures and treatments.

Sen. Lamar Alexander (R-Tenn.) attended the White House announcement and promised to work closely with Obama and Democrats so that cutting-edge medicine begins reaching patients more quickly, while still preserving this nations gold standard for safety and quality The president has recognized this, Chairman Upton in the House is working on this, and I have spoken with [HHS] Secretary [Sylvia Matthews] Burwell about our plans in the Senate health committee to work in a bipartisan way to modernize the way drugs and medical devices are discovered, developed and reviewed.

The development of the genetic consortium has long been a gleam in the eye of NIH director Francis Collins, who led the sequencing of the first human genome at a cost of about $400 million 15 years ago. Now it costs about $1,000 to sequence one persons genes.

Collins said that the lower costs of DNA sequencing and the availability of electronic health records lets scientists do things we couldnt have imagined a decade ago.

Thats one of the reasons were bold enough to say we can do this, he said of the new initiative.

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Obamas precision medicine initiative could bring ...

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Newswise (New York January 30, 2015) Two existing cancer drugs turn on a gene that tells tumor cells to remain inactive, according to a study led by researchers at the Icahn School of Medicine at Mount Sinai and published today in Nature Communications.

Researchers discovered that the gene NR2F1, when switched on, programs tumor cells to stay dormant. When the gene is switched off, tumor cells divide and multiply as part of abnormal growth, potentially allowing dormant cells to grow into tumors throughout the body (metastasis). Combining the anticancer drugs azacytidine and retinoic acid significantly increased the amount of active NR2F1 in tumor cells. These patterns were found in mouse models of several cancers, and confirmed in prostate cancer cells from human patients.

Results suggest that NR2F1 is a master regulator of tumor cell growth, influencing several genes that determine whether cells remain inactive, or quiescent in medical terms. According to the study, NR2F1 exerts control over long lasting programs in stem cells in the human embryo, where it directs cells to stop growing and become specialized cells (neurons) for life. This function suggests that NR2F1 may exert a long-lasting effect on tumor cells, keeping them dormant after they have broken off from an original tumor.

Our results explain why some tumor cells scattered through the body are committed to remaining harmless for years, while others cause active disease, said Julio A. Aguirre-Ghiso, PhD, Professor of Medicine, Hematology and Medical Oncology, and Otolaryngology at the Icahn School of Medicine. In finding this master switch we found a way to analyze tumor cells before treatment to determine the risk of a cancer recurrence or metastasis.

Azacytidine and retinoic acid, the latter a form of vitamin A, prevented tumor cells from rapidly multiplying, restored normal cell function, and activated several tumor suppressor genes that are often turned off in tumors, said study co-leader Maria Soledad Sosa, PhD, a postdoctoral fellow in Hematology at the Icahn School of Medicine. We now have strong evidence that combining these well-known drugs may have a profound, long-lasting therapeutic effect.

The current study builds on the research teams earlier finding that lowering amounts of tumor suppressor genes TGF2 and p38 awakened dormant tumor cells, fueling metastatic tumor growth. Azacytidine and retinoic acid restored TGF2 expression and p38 activation to drive tumor cell dormancy.

This study was supported by grants from the Samuel Waxman Cancer Research Foundation, National Cancer Institute, National Institute of Environmental Health Sciences, New York State Stem Cell Science program, JJR Foundation and Hirschl/Weill-Caulier Trust, Department of Defense and Janssen Research and Development LLC.

About the Mount Sinai Health System The Mount Sinai Health System is an integrated health system committed to providing distinguished care, conducting transformative research, and advancing biomedical education. Structured around seven member hospital campuses and a single medical school, the Health System has an extensive ambulatory network and a range of inpatient and outpatient servicesfrom community-based facilities to tertiary and quaternary care.

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Master Switch Found to Stop Tumor Cell Growth by Inducing Dormancy

Gene publishes papers that focus on the regulation, expression, function and evolution of genes in all biological contexts, including all prokaryotic and eukaryotic organisms, as well as viruses.

Gene strives to be a very diverse journal and topics in all fields will be considered for publication. Although not limited to the following, some general topics include:

DNA Organization, Replication & Evolution -Focus on genomic DNA (chromosomal organization, comparative genomics, DNA replication, DNA repair, mobile DNA, mitochondrial DNA, chloroplast DNA). Expression & Function - Focus on functional RNAs (microRNAs, tRNAs, rRNAs, mRNA splicing, alternative polyadenylation) Regulation - Focus on processes that mediate gene-read out (epigenetics, chromatin, histone code, transcription, translation, protein degradation). Cell Signaling - Focus on mechanisms that control information flow into the nucleus to control gene expression (kinase and phosphatase pathways controlled by extra-cellular ligands, Wnt, Notch, TGFbeta/BMPs, FGFs, IGFs etc.) Profiling of gene expression and genetic variation - Focus on high throughput approaches (e.g., DeepSeq, ChIP-Seq, Affymetrix microarrays, proteomics) that define gene regulatory circuitry, molecular pathways and protein/protein networks. Genetics - Focus on development in model organisms (e.g., mouse, frog, fruit fly, worm), human genetic variation, population genetics, as well as agricultural and veterinary genetics. Molecular Pathology & Regenerative Medicine - Focus on the deregulation of molecular processes in human diseases and mechanisms supporting regeneration of tissues through pluripotent or multipotent stem cells.

Gene encourages submission of novel manuscripts that present a reasonable level of analysis, functional relevance and/or mechanistic insight. Gene also welcomes papers that have predominantly a descriptive component but improve the essential basis of knowledge for subsequent functional studies, or provide important confirmation of recently published discoveries.

The primary criteria for acceptance are that the work is original and scientifically sound. The journal appreciates that standards of novelty are arbitrary, differ among disciplines and geographic locations, as well as change with time. In partnership with Editors, Referees and Authors, the journal will promote the revision of papers to ensure that accepted papers are reasonably complete and competitive with concurrent submissions in a given field.

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An Obama initiative seeks to channel a torrent of gene information into treatments for cancer, other diseases.

President Barack Obama is proposing to spend $215 million on a precision medicine initiative the centerpiece of which will be a national study involving the health records and DNA of one million volunteers, administration officials said yesterday.

Precision medicine refers to treatments tailored to a persons genetic profile, an idea already transforming how doctors fight cancer and some rare diseases.

The Obama plan, including support for studies of cancer and rare disease, is part of a shift away from one-size-fits-all medicine, Jo Handelsman, associate director for the White House Office of Science and Technology Policy, said in a briefing yesterday. She called precision medicine a game changer that holds the potential to revolutionize how we approach health in this country and around the world.

The White House said the largest part of the money, $130 million, would go to the National Institutes of Health in order to create a population-scale study of how peoples genes, environment, and lifestyle affect their health.

According to Francis Collins, director of the National Institutes of Health, the study will recruit new volunteers as well as merging data from several large studies already under way. Details still need to be sorted out, said Collins, but the study could eventually involve completely decoding the genomes of hundreds of thousands of people.

Officials indicated that patients might have more access to data generated about them than is usually the case in research studies. That is partly because scientists will need the ability to re-contact them, should their genes prove interesting.

We arent just talking about research but also about patients access to their own data, so they can participate fully in decisions about their health that affect them, said John Holdren, director of the White House Office of Science and Technology Policy.

The Obama initiative, which the president first announced during his State of the Union address, also allocates $70 million for DNA-driven research on cancer and another $10 million for the U.S. Food and Drug Administration, which has struggled to regulate genome tests.

Collins said the U.S. is not seeking to create a single bio-bank. Instead, the project would look to combine data from among what he called more than 200 large American health studies that are ongoing and together involve at least two million people. Fortunately, we dont have to start from scratch, he said. The challenge of this initiative is to link those together. Its more a distributed approach than centralized.

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U.S. to Develop DNA Study of One Million People

President Obamas $215 million plan to expand research for cancer treatments to fit each individuals genetic makeup could mark a major shift in modern medicine, according to local geneticists.

I think there may be a tipping point soon when people realize genomic medicine is valuable, and this may be part of it, said George Church, professor of genetics at Harvard Medical School. This seems to be part of a grass-roots groundswell that will lead to everyone wanting to have access to their genomic data.

The effort, which Obama called the precision medicine initiative, would study the genes of about a million volunteers to figure out how to personalize treatments for patients instead of using the same approach for each condition.

Doctors have always recognized that every patient is unique, and doctors have always tried to tailor their treatments as best they can to individuals, Obama said yesterday at the White House as he announced the initiative. You can match a blood transfusion to a blood type. That was an important discovery. What if matching a cancer cure to our genetic code was just as easy, just as standard?

The initiative will be in the budget Obama sends to Congress on Monday.

The president said the effort would provide the National Cancer Institute funds to identify genetic factors that cause cancer to help develop sophisticated new treatments. The U.S. Food and Drug Administration would also receive money to evaluate next-generation genetic tests.

Dr. Robert Green, a medical geneticist and physician-scientist at Brigham and Womens Hospital and Harvard Medical School, said the effort is something that the field has yearned for for many years.

Itll be a wonderful accelerator of progress, he said.

He added that while there is some skepticism about whether the field is solid enough now to invest in, this is exactly what is needed to take us to the point where we can start proving the worth of genomic medicine.

Local biotech executives, including from Vertex Pharmaceuticals and Foundation Medicine, were at the White House event.

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Barack Obama eyes gene therapy growth