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Gene Therapy News
February 22, 2017

Latest Headlines
updated 10:03am EST

More Gene Therapy News
February 22, 2017

Research Helps Explain Why Androgen-Deprivation Therapy Doesn't Work for Many Prostate Cancers

Jan. 5, 2017 — Metastatic prostate cancer, or prostate cancer that has spread to other organs, is incurable. In new research, scientists have identified two gatekeeper genes that allow prostate cancer to progress ... read more

Immunotherapy, Gene Therapy Combination Shows Promise Against Glioblastoma

Jan. 4, 2017 — In a new study, gene therapy deployed with immune checkpoint inhibitors demonstrates potential benefit for devastating brain ... read more

Mechanism of Successful Horizontal Gene Transfer Between Divergent Organisms Explained

Dec. 20, 2016 — Researchers have shown how a host's gene regulatory environment can facilitate the establishment of a gene newly arrived via horizontal transfer. Studying the cellulose synthase gene in ... read more

Dec. 14, 2016 — Breast cancer cells that carry a certain gene mutation can be induced to die using a combination of an existing targeted therapy along with an investigational molecule tested by ... read more

Enzyme That Regulates DNA Repair May Offer New Precision Treatments for Breast and Ovarian Cancer

Dec. 12, 2016 — Researchers have identified an enzyme called UCHL3 that regulates the BRCA2 pathway, which is important for DNA repair, report ... read more

Loss of ARID1A Protein Drives Onset, Progress of Colon Cancer

Dec. 12, 2016 — A team of scientists has developed a model system in mice that allows them to look closely at how a protein often mutated in human cancer exerts its tumor-silencing effects. The new findings shed ... read more

After One Dose of Gene Therapy, Hemophilia B Patients Maintain Near-Normal Levels of Clotting Factor

Dec. 3, 2016 — Researchers are reporting the highest and most sustained levels to date of an essential blood-clotting factor IX in patients with the inherited bleeding disorder hemophilia B. After receiving a ... read more

Nov. 30, 2016 — CRISPR/Cas9, a powerful genome editing tool, is showing promise for efficient correction of disease-causing mutations. For the first time, researchers have developed a dual gene therapy approach to ... read more

Successfully Treating Genetically Determined Autoimmune Enteritis

Nov. 28, 2016 — Using targeted immunotherapy, doctors have succeeded in curing a type of autoimmune enteritis caused by a recently discovered genetic mutation. These results raise new possibilities for the ... read more

Pancreatitis Reduced by Nearly 50% After Gene Therapy to Treat Lipoprotein Lipase Deficiency

Nov. 8, 2016 — Over a 6-year period, patients with the genetic disease lipoprotein lipase deficiency (LPLD) who received a single gene therapy treatment of alipogene tiparvovec had a marked reduction in the ... read more

Nov. 8, 2016 — A gene-editing tool known as CRISPR has been used to repair the gene that causes sickle cell disease in human stem cells, which they say is a key step toward developing a gene therapy for the ... read more

Genetic Cause for Shift Work Fatigue Discovered

Nov. 4, 2016 — Some people adapt easily to shift work, but not everyone can handle constant disruptions to their daily rhythm. Researchers have now found that a melatonin receptor gene influences tolerance to shift ... read more

Scientists Show How Mutation Causes Incurable Premature Aging Disease

Nov. 1, 2016 — Scientists have demonstrated how a mutation in a specific protein in stem cells causes an incurable premature aging disease called dyskeratosis congenita, and were able to introduce the mutation into ... read more

Common Heart Problem Caused by Cancer Therapy Avoided Blood Vessel Treatment

Nov. 1, 2016 — Some of the harmful effects of a commonly used cancer drug can be alleviated by using gene therapy that stimulates blood vessel growth in the heart, researchers have ... read more

Oct. 26, 2016 — For the first time, researchers have demonstrated in mice that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1 (NPC1). The gene ... read more

Oct. 26, 2016 — A next-generation gene-editing system has successfully cured a genetic blood disorder in living mice using a simple IV treatment. Unlike the popular CRISPR gene-editing technique, the new technology ... read more

Oct. 26, 2016 — A new gene editing strategy has been used to correct mutations that cause thalassemia, a form of anemia. Their gene editing technique provided corrections to the mutations and alleviated the disease ... read more

In Crafting New Treatments for Hemophilia, a 'Less Is More' Approach

Oct. 17, 2016 — Hematology researchers have found that blocking the role of a common protein may offer unexpected benefits for patients with the inherited bleeding disorder hemophilia A. The finding offers potential ... read more

Oct. 4, 2016 — The associations with type 2 diabetes and coronary artery disease of low-density lipoprotein cholesterol (LDL-C)-lowering genetic variants have been the focus of recent ... read more

Sep. 21, 2016 — Gene therapy is a kind of experimental treatment that is designed to fix faulty genetic material and help a patient fight off or recover from a disease. Now scientists have engineered the ... read more

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