Gene Therapy News
September 3, 2015

Latest Headlines
updated 11:30am EDT

More Gene Therapy News
September 3, 2015

Aug. 12, 2015 — Heteroduplex oligonucleotide is a brand new oligonucleotide drug. It is found to be significantly potent at reducing expression of the target RNA, and also improves the phenotype in disease models ... read more

Gene Modulation Method May Provide Insight on Regrowing Inner-Ear Sensory Hair Cells

June 11, 2015 — An expert in the biology and physiology of the inner ear has developed a method to temporally modify the expression of the retinoblastoma-1 gene in mice. Modulation of the RB1 gene can allow for the ... read more

Night Blindness: Finding Hope in the Dark

June 10, 2015 — A gene that could be responsible for some cases of human night blindness has been discovered by researchers. Leber's congenital amaurosis (LCA) is a group of hereditary retinal diseases that ... read more

Gene Therapy, Surgery Could Mean Eight More Months for Sickest Brain Cancer Patients

May 31, 2015 — As the most malignant of primary brain cancers, glioblastomas are frustratingly difficult to treat. Surgery is often risky and cannot remove all of the tumor, and chemotherapy eventually becomes ... read more

Genetic Defect Linked to Visual Impairment in Dyslexics

May 26, 2015 — A risk gene for dyslexia is associated with impairments in visual motion detection, according to a study. Mutations in the gene DCDC2 have previously been associated with dyslexia, and this study ... read more

Therapy-Resistant Breast Cancer Mechanism Revealed

May 26, 2015 — A cluster of defined, non-coding RNAs are mechanistically involved in endocrine therapy resistance in human breast cancer cells, new research has revealed. Furthermore, resveratrol, a kind of ... read more

New Age of Genome Editing Could Lead to Cure for Sickle Cell Anemia

May 14, 2015 — Researchers have shown that changing just a single letter of the DNA of human red blood cells in the laboratory increases their production of oxygen-carrying hemoglobin -- a world-first advance that ... read more

X-Linked Gene Mutations Cause Some Cases of Male Infertility, Study Says

May 13, 2015 — Some cases of male infertility are due to mutations in the maternal X chromosome that prevent development of viable sperm, according to a study. Nearly half of cases of male infertility not due to a ... read more

Androgen Deprivation Therapy May Lead to Cognitive Impairment in Prostate Cancer Patients

May 13, 2015 — Cognitive impairment can occur in cancer patients who are treated with a variety of therapies, including radiation therapy, hormone therapy, and chemotherapy. After chemotherapy treatment it is ... read more

May 11, 2015 — Even with the best available treatments, the median survival of patients with metastatic, hormone-refractory prostate cancer is only two to three years. Driven by the need for more effective ... read more

May 5, 2015 — A gene variant that influences whether Huntington’s disease breaks out earlier or later than expected has been identified by researchers. The findings can contribute to improved diagnosis and ... read more

May 4, 2015 — A closer look at the DNA around the Huntington's disease gene offers researchers a new understanding of how the gene is controlled and how this affects the disease. These findings set the stage ... read more

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

Apr. 29, 2015 — Cardiomyopathies are diseases of weakened heart muscle, which can lead to heart enlargement and heart failure. The conditions are often genetically inherited. Gene therapy can clip out genetic ... read more

Gene Editing Used to Correct Mutation in Cystic Fibrosis

Apr. 27, 2015 — Researchers have successfully corrected the most common mutation in the gene that causes cystic fibrosis. Cystic fibrosis is an inherited, life-threatening disorder that damages the lungs and ... read more

Toxic Mushroom-Based Drug May Help Battle Colorectal Cancer

Apr. 22, 2015 — For some time, cancer scientists have considered the toxin, alpha-amanatin derived from “death cap” mushrooms, as a possible cancer treatment. However, due to its penchant for causing liver ... read more

New Gene Therapy Success in a Rare Disease of the Immune System

Apr. 21, 2015 — The efficacy of gene therapy treatment for Wiskott-Aldrich syndrome has been demonstrated by researchers. Wiskott-Aldrich syndrome is a rare congenital immune and platelet deficiency which is ... 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

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

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

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