Contact Information
Available for logged-in reporters only
Newswise LA JOLLA, CA June 26, 2014 Scientists working to make gene therapy a reality have solved a major hurdle: how to bypass a blood stem cells natural defenses and efficiently insert disease-fighting genes into the cells genome.
In a new study led by Associate Professor Bruce Torbett at The Scripps Research Institute (TSRI), a team of researchers report that the drug rapamycin, which is commonly used to slow cancer growth and prevent organ rejection, enables delivery of a therapeutic dose of genes to blood stem cells while preserving stem cell function.
These findings, published recently online ahead of print by the journal Blood, could lead to more effective and affordable long-term treatments for blood cell disorders in which mutations in the DNA cause abnormal cell functions, such as in leukemia and sickle cell anemia.
Improving Gene Delivery to Blood Stem Cells
Viruses infect the body by inserting their own genetic material into human cells. In gene therapy, however, scientists have developed gutted viruses, such as the human immunodeficiency virus (HIV), to produce what are called viral vectors. Viral vectors carry therapeutic genes into cells without causing viral disease. Torbett and other scientists have shown that HIV vectors can deliver genes to blood stem cells.
For a disease such as leukemia or leukodystrophy, where mutations in the DNA cause abnormal cell function, efficiently targeting the stem cells that produce these blood cells could be a successful approach to halting the disease and prompting the body to produce healthy blood cells.
If you produce a genetic modification in your blood stem cells when you are five years old, these changes are lifelong, said Torbett. Furthermore, the gene-modified stem cells can develop into many types of cells that travel throughout the body to provide therapeutic effects.
However, because cells have adapted defense mechanisms to overcome disease-causing viruses, engineered viral vectors can be prevented from efficiently delivering genes. Torbett said that when scientists extract blood stem cells from the body for gene therapy, HIV viral vectors are usually able to deliver genes to only 30 to 40 percent of them. For leukemia, leukodystrophy or genetic diseases where treatment requires a reasonable number of healthy cells coming from stem cells, this number may be too low for therapeutic purposes.
Go here to see the original:
Scripps Research Institute Scientists Find Potential New Use for Cancer Drug in Gene Therapy for Blood Disorders
- Gene Therapy: Ethical Issues: Information from Answers.com - February 6th, 2014 [February 6th, 2014]
- Gene, immune therapy help cancer war - April 10th, 2014 [April 10th, 2014]
- Gene Therapy - Genetics Home Reference - Your guide to ... - April 10th, 2014 [April 10th, 2014]
- What is gene therapy? - Genetics Home Reference - April 10th, 2014 [April 10th, 2014]
- Gene therapy - Wikipedia, the free encyclopedia - April 10th, 2014 [April 10th, 2014]
- Gene Therapy PSA Dan Lesser Period 2 - Video - April 15th, 2014 [April 15th, 2014]
- Gene therapy basics - Video - April 15th, 2014 [April 15th, 2014]
- In vivo gene therapy - Video - April 15th, 2014 [April 15th, 2014]
- Ex vivo gene therapy - Video - April 15th, 2014 [April 15th, 2014]
- Broad Institute Gets Patent on Revolutionary Gene-Editing Method - April 17th, 2014 [April 17th, 2014]
- Gene Therapy - Video - April 17th, 2014 [April 17th, 2014]
- Mark Hamalainen: Gene Therapy - Video - April 17th, 2014 [April 17th, 2014]
- JFK - Gene Therapy for Brain Tumors - Video - April 18th, 2014 [April 18th, 2014]
- Fixing the Liver with New Gene Therapy (Brainstorm Ep178) - Video - April 18th, 2014 [April 18th, 2014]
- Gene Therapy | Buzzle.com - April 22nd, 2014 [April 22nd, 2014]
- Gene Therapy I - RCN Corporation - April 22nd, 2014 [April 22nd, 2014]
- Ricky Ashby Microbiology Podcast - Video - April 22nd, 2014 [April 22nd, 2014]
- Regrown nerves boost bionic ears - April 24th, 2014 [April 24th, 2014]
- Cochlear Implant Plus Gene Therapy Could Restore Hearing to the Deaf - April 24th, 2014 [April 24th, 2014]
- Scientists reverse memory loss in mice with Alzheimer's - April 24th, 2014 [April 24th, 2014]
- Bionic Ears Boosted by Gene Therapy and Regrown Nerves - April 24th, 2014 [April 24th, 2014]
- Study: Gene Therapy May Boost Cochlear Implants - April 24th, 2014 [April 24th, 2014]
- Gene therapy may boost hearing, study finds - April 24th, 2014 [April 24th, 2014]
- Pronounce Medical Words Gene Therapy - Video - April 24th, 2014 [April 24th, 2014]
- Gene therapy may boost power of cochlear implants, study says - April 25th, 2014 [April 25th, 2014]
- Gene therapy shows promise to help people regrow auditory nerve cells - April 25th, 2014 [April 25th, 2014]
- Cochlear Implant Also Uses Gene Therapy to Improve Hearing - April 25th, 2014 [April 25th, 2014]
- Gene Therapy May Enhance Cochlear Implants, Animal Study Finds - April 25th, 2014 [April 25th, 2014]
- Uniqure Gene Therapy Info. - Video - April 25th, 2014 [April 25th, 2014]
- Jean Bennett on gene therapy as a treatment for blindness - Video - April 25th, 2014 [April 25th, 2014]
- mice-science-AFPrelax-250414.jpg - April 26th, 2014 [April 26th, 2014]
- Cochlear implant enhances patient experience through gene therapy - April 28th, 2014 [April 28th, 2014]
- Gene therapy may help hearing - April 28th, 2014 [April 28th, 2014]
- Ethics of Gene therapy - Video - April 30th, 2014 [April 30th, 2014]
- Novel Financing For Gene Therapy Company - May 1st, 2014 [May 1st, 2014]
- Gene Therapy To Enhance Hearing - ABC Austrlia News - 2014 - - Video - May 1st, 2014 [May 1st, 2014]
- Biotechnology - GMO's & Gene Therapy - Video - May 1st, 2014 [May 1st, 2014]
- Cochlear Implant Gets an Upgrade with Gene Therapy - Video - May 4th, 2014 [May 4th, 2014]
- Gene therapy for haemophilia - Professor Amit Nathwani - Video - May 4th, 2014 [May 4th, 2014]
- Retrovirus in Gene Therapy - Video - May 4th, 2014 [May 4th, 2014]
- Regeneron, Avalanche To Develop Novel Gene Therapy Products In Ophthalmology - May 5th, 2014 [May 5th, 2014]
- Gene Therapy Used to Preserve Sight in Patients - May 5th, 2014 [May 5th, 2014]
- SCID gene therapy class - Video - May 6th, 2014 [May 6th, 2014]
- Gene Therapy using SMaRT - Video - May 6th, 2014 [May 6th, 2014]
- gene therapy video - Video - May 9th, 2014 [May 9th, 2014]
- Chronic Granulomatous Disease (CGD) - Video - May 12th, 2014 [May 12th, 2014]
- Gene Therapy (Breast Cancer) - Video - May 12th, 2014 [May 12th, 2014]
- Suicide gene therapy - Video - May 12th, 2014 [May 12th, 2014]
- Employment of gene therapy in depression (psychiatric disorder) - Video - May 12th, 2014 [May 12th, 2014]
- Gene therapy for blindness (2012) - Video - May 12th, 2014 [May 12th, 2014]
- AGRC1020 Video Presentation Gene Therapy restores colour vision in monkeys - Video - May 12th, 2014 [May 12th, 2014]
- gene therapy~3 - Video - May 12th, 2014 [May 12th, 2014]
- Gene Delivery System (In-vivo and Ex-vivo) HD - Video - May 12th, 2014 [May 12th, 2014]
- Spark Therapeutics to open headquarters in West Philadelphia - May 14th, 2014 [May 14th, 2014]
- Gene Therapy - Nature - May 14th, 2014 [May 14th, 2014]
- Gene therapy for RTT syndrome benefits and drawbacks - Video - May 16th, 2014 [May 16th, 2014]
- Gene Therapy (Using Biolistic gene gun for gene delivery) - Video - May 16th, 2014 [May 16th, 2014]
- Germline Gene Therapy - Video - May 16th, 2014 [May 16th, 2014]
- Gene Therapy Biology Project for Swavely - Video - May 16th, 2014 [May 16th, 2014]
- Gene therapy extends survival in an animal model of spinal muscular atrophy - May 22nd, 2014 [May 22nd, 2014]
- Novel RNAi therapy silences mutated Huntington's disease gene and reduces symptoms - May 22nd, 2014 [May 22nd, 2014]
- Global Gene Therapy Market: Analysis, Size, Share, Growth, Trends and Forecast 2013 - 2019 - May 22nd, 2014 [May 22nd, 2014]
- Gene therapy hits blood cancers hard - May 22nd, 2014 [May 22nd, 2014]
- Spark Therapeutics late-stage gene therapy for inherited blindness takes a $73M step forward - May 31st, 2014 [May 31st, 2014]
- Genome Editing to Reverse Bubble Boy Syndrome - May 31st, 2014 [May 31st, 2014]
- Genetic Researching: Gene Therapy - Video - May 31st, 2014 [May 31st, 2014]
- BEYOND THE BUBBLE: 10 Years of Gene Therapy - Video - May 31st, 2014 [May 31st, 2014]
- Gene Therapy Combined with IMRT Found to Reduce the Rate of Positive Prostate Biopsy After Treatment for Intermediate ... - June 4th, 2014 [June 4th, 2014]
- Gene therapy combined with IMRT found to reduce recurrence for select prostate cancer patients - June 4th, 2014 [June 4th, 2014]
- Gene therapy combined with IMRT reduces rate of positive prostate biopsy after treatment for intermediate-risk ... - June 4th, 2014 [June 4th, 2014]
- Bluebird, Agios: Preview of Next Week's Important Pipeline Updates - June 12th, 2014 [June 12th, 2014]
- Attack of the B - Team EP8 - Drowning for Science! - Video - June 12th, 2014 [June 12th, 2014]
- 3rd International Conference and Exhibition on Cell & Gene Therapy - Video - June 14th, 2014 [June 14th, 2014]
- Blood Disorder Patients Respond Strongly to Bluebird Bio Gene Therapy - June 15th, 2014 [June 15th, 2014]
- Gene Therapy Partnering Terms and Agreements - Video - June 15th, 2014 [June 15th, 2014]
- Gene Therapy - GMAT Free - GFSC120 - Video - June 18th, 2014 [June 18th, 2014]
- New Gene Therapy Rapidly Helps Patients With Rare Blood Disorder - Video - June 18th, 2014 [June 18th, 2014]
- Isis Could Get a $2 Billion Gain From Heart Attack Trials - June 22nd, 2014 [June 22nd, 2014]
- Gene Therapy for Beta Thalassemia - Video - June 22nd, 2014 [June 22nd, 2014]
- A level. R.7. Cystic fibrosis and gene therapy (Ms Cooper) - Video - June 24th, 2014 [June 24th, 2014]