Gene Medicine Xagena
The FDA ( Food and Drug Administration ) issued a historic action making the first gene therapy available in the United States, ushering in a new approach to the treatment of cancer and other serious ...
Increased secretion of growth hormone leads to gigantism in children and acromegaly in adults; the genetic causes of gigantism and acromegaly are poorly understood. Researchers performed clinical a ...
Parkinson's disease is typically treated with oral dopamine replacement therapies; however, long-term treatment leads to motor complications and, occasionally, impulse control disorders caused by inte ...
Choroideremia is an X-linked recessive disease that leads to blindness due to mutations in the CHM gene, which encodes the Rab escort protein 1 ( REP1 ). The effects of retinal gene therapy with an a ...
Corneal graft rejection is a major problem in chronic herpetic keratitis (HK ) patients with latent infection. A new class of antiviral agents targeting latent and active forms of herpes simplex viru ...
National Institutes of Health ( NHI ) researchers have identified gene variants that cause a rare syndrome of sporadic fevers, skin rashes and recurring strokes, beginning early in childhood. The disc ...
The St. Jude Children's Research Hospital-Washington University Pediatric Cancer Genome Project has identified the most common genetic alteration ever reported in the brain tumor ependymoma and the ev ...
After a myocardial infarction, there is often permanent damage to a portion of the heart. This happens, in part, because cardiac muscle cells are terminally differentiated and cannot proliferate after ...
Researchers from the University of Washington and the HudsonAlpha Institute for Biotechnology have developed a new method for organizing and prioritizing genetic data. The Combined Annotation-Dependen ...
Nerve growth factor ( NGF ) is an endogenous neurotrophic-factor protein with the potential to restore function and to protect degenerating cholinergic neurons in Alzheimer's disease, but safe and eff ...
Choroideremia is an X-linked recessive disease that leads to blindness due to mutations in the CHM gene, which encodes the Rab escort protein 1 ( REP1 ). Researchers have assessed the effects of retin ...
Researchers have developed a novel strategy for treatment of Leber hereditary optic neuropathy ( LHON ) caused by a mutation in the nicotinamide adenine dinucleotide dehydrogenase subunit IV ( ND4 ) m ...
Successful gene therapy largely depends on the selective introduction of therapeutic genes into the appropriate target cancer cells. One of the most effective and promising approaches for targeting tu ...
Nonviral gene therapy represents a realistic option for clinical application in cancer treatment. A preclinical study has demonstrated the advantage of using the small-size MIDGE DNA vector for improv ...
Progranulin protein ( PGRN ) is a cysteine-rich growth factor encoded by the progranulin gene ( GRN ). PGRN mutations were identified in patients with frontotemporal lobar degeneration ( FTLD ) and re ...
