Gene Therapy News
May 25, 2015

Latest Headlines
updated 12:59am EDT

More Gene Therapy News
May 25, 2015

Gene Therapy Efficacy for Leber Congenital Amaurosis Is Dynamic: Improvement Is Followed by Decline in Vision

May 3, 2015 — Gene therapy for Leber congenital amaurosis (LCA), an inherited disorder that causes loss of night- and day-vision starting in childhood, improved patients’ eyesight within weeks of treatment in a ... read more

Novel Neurodegenerative Disease and Gene Identified With the Help of 'Man's Best Friend'

Apr. 16, 2015 — A breakthrough study performed with veterinary neurologists and neuropathologists has identified a gene mutation that causes a novel type of neurodegenerative disease in dogs. The results of the ... read more

New Genetic Mutation Could Signal Start of Malaria Drug Resistance in Africa

Apr. 17, 2015 — Early indicators of the malaria parasite in Africa developing resistance to the most effective drug available have been confirmed, according to new research. Investigators found Plasmodium falciparum ... read more

Gene Therapy Superior to Half-Matched Transplant for 'Bubble Boy Disease'

Apr. 13, 2015 — Children with 'bubble boy disease' who undergo gene therapy have fewer infections and hospitalizations than those receiving stem cells from a partially matched donor, new research suggests. ... read more

Apr. 3, 2015 — Scientists have identified a small RNA molecule that can suppress cancer-causing genes in mice with glioblastoma mulitforme, a deadly and incurable type of brain tumor. While standard chemotherapy ... read more

Like Angelina Jolie, Study Pinpoints Genetic Cause of Increased Leukemia Risk

Mar. 25, 2015 — A newly-discovered, heritable genetic cause of acute lymphoblastic leukemia (ALL), namely mutation of the gene ETV6, has been discovered by researchers. "These people are born with a broken gene ... read more

Mar. 23, 2015 — Erectile dysfunction is a taboo subject among men. No one likes to talk about it. But the fact is that as men age, an increasing number will suffer from erectile dysfunction. From the age of 30, the ... read more

Consistency Is the Key to Success in Bread Baking, Biology

Mar. 16, 2015 — Whether you're baking bread or building an organism, the key to success is consistently adding ingredients in the correct order and in the right amounts, according to a new genetic ... read more

Boosting a Natural Protection Against Alzheimer's Disease

Mar. 12, 2015 — A gene variant has been identified by researchers that may be used to predict people most likely to respond to an investigational therapy under development for Alzheimer's disease (AD). The ... read more

Mar. 12, 2015 — Researchers have been able to grow patient-derived, healthy cells in the lab, coming a step closer to treating fatal blood disorders. "This work demonstrates a new method that could lead to a ... read more

Study Bolsters 'Turbocharged' Protein as a Promising Tool in Hemophilia Gene Therapy

Mar. 12, 2015 — Using gene therapy to produce a mutant human protein with unusually high blood-clotting power, scientists have successfully treated dogs with the bleeding disorder hemophilia, without triggering an ... read more

Mar. 12, 2015 — A reprogrammed retrovirus could successfully transfer new clotting genes into animals with hemophilia B to safely and dramatically decrease spontaneous bleeding, a new study has ... read more

Cancer Treatment: New Nanodevice Defeats Drug Resistance

Mar. 2, 2015 — Chemotherapy often shrinks tumors at first, but as cancer cells become resistant to drug treatment, tumors can grow back. A new nanodevice can help overcome that by first blocking the gene that ... read more

Mutation in APC2 Gene Causes Sotos Features

Mar. 5, 2015 — Sotos syndrome is a congenital syndrome that is characterized by varying degrees of mental retardation and a large head circumference etc. It is known that 90 percent of Sotos syndrome patients have ... read more

Genetic Pathways Linked to CF Disease Severity Pinned Down

Feb. 23, 2015 — Mutation of one gene is all it takes to get cystic fibrosis, but disease severity depends on many other genes and proteins. For the first time, researchers identified genetic pathways that play major ... read more

Feb. 18, 2015 — A novel gene-delivery system that shuttles a gene into a cell, but only for a temporary stay, has been developed by researchers, providing a potential new gene-therapy strategy for treating disease. ... read more

Serendipity Leads to Discovery of Adult Cancer Genes Driving Young-Adult Ewing Sarcoma

Feb. 3, 2015 — A new study finds alterations in expression of genes PIK3R3 and PTEN, more commonly observed in adult tumors, in the rare, young-adult bone cancer Ewing Sarcoma, potentially offering ways to improve ... read more

New Nanoparticle Gene Therapy Strategy Effectively Treats Deadly Brain Cancer in Rats

Feb. 4, 2015 — Nanoparticles have been used to successfully deliver a new therapy to cancer cells in the brains of rats, prolonging their lives, scientists report. Previous research on mice found that nanoparticles ... read more

Testing for EGFR Mutations, ALK Rearrangements Is Cost-Effective in NSCLC.

Jan. 29, 2015 — Multiplexed genetic screening for epidermal growth factor receptor and anaplastic lymphoma kinase gene rearrangements and subsequent biomarker-guided treatment is cost-effective compared with ... read more

Parkinson's Gene Linked to Lung Cancer

Jan. 29, 2015 — A gene that is associated with lung cancer has been identified by researchers. Through whole exome sequencing, they identified a link between a mutation in PARK2, a gene associated with early-onset ... read more

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