Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, and hereditary diseases in which a defective mutant allele is replaced with a functional one.
Although the technology is still in its infancy, it has been used with some success.
Antisense therapy is not strictly a form of gene therapy, but is a genetically-mediated therapy and is often considered together with other methods.
In most gene therapy studies, a "normal" gene is inserted into the genome to replace an "abnormal," disease-causing gene.
A carrier called a vector must be used to deliver the therapeutic gene to the patient's target cells.
Currently, the most common type of vectors are viruses that have been genetically altered to carry normal human DNA.
Viruses have evolved a way of encapsulating and delivering their genes to human cells in a pathogenic manner.
Scientists have tried to harness this ability by manipulating the viral genome to remove disease-causing genes and insert therapeutic ones.
Target cells such as the patient's liver or lung cells are infected with the vector.
The vector then unloads its genetic material containing the therapeutic human gene into the target cell.
The generation of a functional protein product from the therapeutic gene restores the target cell to a normal state.
In theory it is possible to transform either somatic cells (most cells of the body) or cells of the germline (such as sperm cells, ova, and their stem cell precursors).
All gene therapy to date on humans has been directed at somatic cells, whereas germline engineering in humans remains controversial.
For the introduced gene to be transmitted normally to offspring, it needs not only to be inserted into the cell, but also to be incorporated into the chromosomes by genetic recombination.
Somatic gene therapy can be broadly split in to two categories: ex vivo, which means exterior (where cells are modified outside the body and then transplanted back in again) and in vivo, which means interior (where genes are changed in cells still in the body).
Recombination-based approaches in vivo are especially uncommon, because for most DNA constructs recombination has a very low probability.
See the original post:
- About the Gene Therapy Review [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Contribute an Article [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- EBSCO Publishing Deal [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Advertising Opportunities [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Instructions for Authors [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Isis Collaboration With Ortho-McNeil Inc for Metabolic Diseases [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Dystrophin Gene Transfer safe in Duchenne muscular dystrophy [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Researchers Identify Gene for Rare Form of Spinal Muscular Atrophy [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Fatal brain cancer tamed by New gene therapy [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Gene therapy effective in fighting obesity in mice [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Genzyme gene therapy for people with peripheral artery disease failed in a clinical trial to help them regain some mobility [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Gene Therapy May Stall Inherited Emphysema [Last Updated On: December 31st, 2009] [Originally Added On: December 31st, 2009]
- Gene Therapy and Stem Cells Save Limb [Last Updated On: December 31st, 2009] [Originally Added On: December 31st, 2009]
- Faulty Circuits (preview) [Last Updated On: August 17th, 2024] [Originally Added On: April 7th, 2010]
- Rare flowers and common herbal supplements get unmasked with plant DNA barcoding [Last Updated On: August 17th, 2024] [Originally Added On: April 20th, 2010]
- Biomarker Studies Could Realize Goal of More Effective and Personalized Cancer Medicine [Last Updated On: August 17th, 2024] [Originally Added On: April 27th, 2010]
- Schizophrenia shares genetic links with autism, genome study shows [Last Updated On: August 17th, 2024] [Originally Added On: May 12th, 2010]
- Alzheimer's: Forestalling the Darkness with New Approaches (preview) [Last Updated On: August 17th, 2024] [Originally Added On: May 28th, 2010]
- Alzheimer's: Forestalling the Darkness with New Approaches (preview) [Last Updated On: August 17th, 2024] [Originally Added On: May 28th, 2010]
- Large-Scale Autism Study Reveals Disorder's Genetic Complexity [Last Updated On: August 17th, 2024] [Originally Added On: June 12th, 2010]
- Large-Scale Autism Study Reveals Disorder's Genetic Complexity [Last Updated On: August 17th, 2024] [Originally Added On: June 12th, 2010]
- Cancer Therapy Goes Viral: Progress Is Made Tackling Tumors with Viruses [Last Updated On: August 17th, 2024] [Originally Added On: June 24th, 2010]
- Vaccines Derived from Patients' Tumor Cells Are Individualizing Cancer Treatment [Last Updated On: August 17th, 2024] [Originally Added On: June 26th, 2010]
- Vaccines Derived from Patients' Tumor Cells Are Individualizing Cancer Treatment [Last Updated On: August 17th, 2024] [Originally Added On: June 26th, 2010]
- A genome story: 10th anniversary commentary by Francis Collins [Last Updated On: August 17th, 2024] [Originally Added On: June 29th, 2010]
- Hair Trigger: How a Cell's Primary Cilium Functions as a Molecular Antenna [Last Updated On: August 17th, 2024] [Originally Added On: June 30th, 2010]
- Hair Trigger: How a Cell's Primary Cilium Functions as a Molecular Antenna [Last Updated On: August 17th, 2024] [Originally Added On: June 30th, 2010]
- DNA Drugs Come of Age (preview) [Last Updated On: August 17th, 2024] [Originally Added On: July 16th, 2010]
- 2 Genes Linked to Embryonic Brain Impairment in Down's Syndrome [Last Updated On: August 17th, 2024] [Originally Added On: July 22nd, 2010]
- 2 Genes Linked to Embryonic Brain Impairment in Down's Syndrome [Last Updated On: August 17th, 2024] [Originally Added On: July 22nd, 2010]
- Stem Cells from Reprogrammed Adult Cells Found to Bring Along Genetic Defects of Their Donors [Last Updated On: August 17th, 2024] [Originally Added On: October 11th, 2010]
- Was Darwin a Punk? A Q&A with Punker-Paleontologist Greg Graffin [Last Updated On: August 17th, 2024] [Originally Added On: October 11th, 2010]
- Was Darwin a Punk? A Q&A with Punker-Paleontologist Greg Graffin [Last Updated On: August 17th, 2024] [Originally Added On: October 11th, 2010]
- Parkinsonian Power Failure: Neuron Degeneration May Be Caused by a Cellular Energy System Breakdown [Last Updated On: August 17th, 2024] [Originally Added On: October 11th, 2010]
- Desperation Drives Parents to Dubious Autism Treatments (preview) [Last Updated On: August 17th, 2024] [Originally Added On: October 17th, 2010]
- Revolution Postponed: Why the Human Genome Project Has Been Disappointing (preview) [Last Updated On: August 17th, 2024] [Originally Added On: November 7th, 2010]
- Controlling the Brain with Light (preview) [Last Updated On: August 17th, 2024] [Originally Added On: November 7th, 2010]
- Optogenetics: Controlling the Brain with Light [Extended Version] [Last Updated On: August 17th, 2024] [Originally Added On: November 7th, 2010]
- Clear New Insights into the Genetics of Depression [Last Updated On: August 17th, 2024] [Originally Added On: November 7th, 2010]
- TEDMED 2010: Technology and the people [Last Updated On: August 17th, 2024] [Originally Added On: November 7th, 2010]
- Bacteria, the anti-cancer soldier [Last Updated On: August 17th, 2024] [Originally Added On: November 7th, 2010]
- Scientific regress: When science goes backward [Last Updated On: August 17th, 2024] [Originally Added On: February 14th, 2011]
- Can You Live Forever? Maybe Not--But You Can Have Fun Trying [Last Updated On: August 17th, 2024] [Originally Added On: February 14th, 2011]
- How to Fix the Obesity Crisis (preview) [Last Updated On: August 17th, 2024] [Originally Added On: February 14th, 2011]
- Personalizing cancer medicine [Last Updated On: August 17th, 2024] [Originally Added On: February 14th, 2011]
- New Salmonella strain delivers gene-based therapy to fight virus in mice [Last Updated On: August 17th, 2024] [Originally Added On: February 14th, 2011]
- Steps toward a Bionic Eye [Last Updated On: August 17th, 2024] [Originally Added On: February 20th, 2011]
- Giving HIV a Poor Reception: New AIDS Treatment Tinkers with Immune Cell Genes [Last Updated On: August 17th, 2024] [Originally Added On: March 6th, 2011]
- Smaller, cheaper, faster: Does Moore's law apply to solar cells? [Last Updated On: August 17th, 2024] [Originally Added On: March 27th, 2011]
- Smaller, cheaper, faster: Does Moore's law apply to solar cells? [Last Updated On: August 17th, 2024] [Originally Added On: March 27th, 2011]
- New Drugs for Hepatitis C on the Horizon [Last Updated On: August 17th, 2024] [Originally Added On: April 10th, 2011]
- Can we capture all of the world's carbon emissions? [Last Updated On: August 17th, 2024] [Originally Added On: April 10th, 2011]
- Can we capture all of the world's carbon emissions? [Last Updated On: August 17th, 2024] [Originally Added On: April 10th, 2011]
- Drug-resistant genes found in cholera and dysentery strains in New Delhi water supply [Last Updated On: August 17th, 2024] [Originally Added On: May 1st, 2011]
- Fast Track to Vaccines: How Systems Biology Speeds Drug Development (preview) [Last Updated On: August 17th, 2024] [Originally Added On: May 1st, 2011]
- TNVitamins.com – $10 Off Of $50 order [Last Updated On: May 7th, 2011] [Originally Added On: May 7th, 2011]
- 15% Off Any PetAlive Order [Last Updated On: May 7th, 2011] [Originally Added On: May 7th, 2011]
- At PetAlive – $10 off order of $50 or more [Last Updated On: May 7th, 2011] [Originally Added On: May 7th, 2011]
- Native Remedies coupon – 5% Off Any Order [Last Updated On: May 7th, 2011] [Originally Added On: May 7th, 2011]
- Native Remedies – Save $5 coupon [Last Updated On: May 7th, 2011] [Originally Added On: May 7th, 2011]
- Welcome to the Gene Therapy Review [Last Updated On: May 15th, 2011] [Originally Added On: May 15th, 2011]
- Editorial Board [Last Updated On: May 15th, 2011] [Originally Added On: May 15th, 2011]
- Gene Therapy Job Board [Last Updated On: May 15th, 2011] [Originally Added On: May 15th, 2011]
- Corporate Membership [Last Updated On: May 22nd, 2011] [Originally Added On: May 22nd, 2011]
- Native Remedies coupon – 25% Off Any Order [Last Updated On: May 29th, 2011] [Originally Added On: May 29th, 2011]
- What is Gene Therapy? [Last Updated On: June 19th, 2011] [Originally Added On: June 19th, 2011]
- Autism's Tangled Genetics Full of Rare and Varied Mutations [Last Updated On: August 17th, 2024] [Originally Added On: June 19th, 2011]
- Autism's Tangled Genetics Full of Rare and Varied Mutations [Last Updated On: August 17th, 2024] [Originally Added On: June 19th, 2011]
- A New Look at Obsessive-Compulsive Disorder (preview) [Last Updated On: August 17th, 2024] [Originally Added On: June 19th, 2011]
- Close Encounters of Science and Medicine [Last Updated On: August 17th, 2024] [Originally Added On: July 3rd, 2011]
- New Report Details Uphill Battle to Solve the U.S.'s Pain Problem [Last Updated On: August 17th, 2024] [Originally Added On: July 24th, 2011]
- New Report Details Uphill Battle to Solve the U.S.'s Pain Problem [Last Updated On: August 17th, 2024] [Originally Added On: July 24th, 2011]
- A Breath of Fresh Air: New Hope for Cystic Fibrosis Treatment (preview) [Last Updated On: August 17th, 2024] [Originally Added On: August 7th, 2011]
- Studying Mental Illness in a Dish [Last Updated On: August 17th, 2024] [Originally Added On: November 13th, 2011]
- The Puzzle of Pancreatic Cancer: How Steve Jobs Did Not Beat the Oddsbut Nobel Winner Ralph Steinman Did [Last Updated On: August 17th, 2024] [Originally Added On: November 13th, 2011]
- The Puzzle of Pancreatic Cancer: How Steve Jobs Did Not Beat the Odds?but Nobel Winner Ralph Steinman Did [Last Updated On: August 17th, 2024] [Originally Added On: November 13th, 2011]
- Did Alternative Medicine Extend or Abbreviate Steve Jobs's Life? [Last Updated On: August 17th, 2024] [Originally Added On: November 13th, 2011]
- Did Alternative Medicine Extend or Abbreviate Steve Jobs's Life? [Last Updated On: August 17th, 2024] [Originally Added On: November 13th, 2011]
- Calendar: MIND Events in November and December [Last Updated On: August 17th, 2024] [Originally Added On: November 13th, 2011]
- He's No Gregory House--Which Is a Good Thing (preview) [Last Updated On: August 17th, 2024] [Originally Added On: November 20th, 2011]