The Future of Gene Therapy

In cortical neurons, hypoxia-inducible factor 1 (HIF-1) attenuates amyloid-beta protein neurotoxicity and decreases apoptosis induced by oxidative stress or hypoxia.

Prof. Xiqing Chai and co-workers from Hebei Chemical and Pharmaceutical College, China constructed a recombinant adeno-associated virus (rAAV) vector expressing the human HIF-1α gene (rAAV-HIF-1α) efficiently, and tested the assumption that rAAV-HIF-1α represses hippocampal neuronal apoptosis induced by amyloid-beta protein.

Researchers Develop Patient-Specific Stem Cells and Personalized Gene Therapy
Researchers from Columbia University Medical Center (CUMC) have created a way to develop personalized gene therapies for patients with retinitis pigmentosa (RP), a leading cause of vision loss.

Their results confirmed that rAAV-HIF-1α significantly reduces apoptosis induced by amyloid-beta protein in primary cultured hippocampal neurons. This mechanism may be related to HIF-1α-decreased hippocampal neuronal intracellular calcium concentration ([Ca2+]i), thereby inhibiting apoptotic cascade reactions.

With these investigations published in the Neural Regeneration Research (Vol. 9, No. 11, 2014), gene therapy using rAAV to deliver HIF-1α may ultimately provide a new option for clinical treatment of Alzheimer's disease.

Source-Eurekalert

Regeneration of Retinal Ganglion Cell Axons and Gene Therapy
The adult mammalian central nervous system has only limited intrinsic capacity to regenerate connections after injury.

Cellular Mechanism Involved in Memory Consolidation may Develop Gene Therapy
The first cause of dementia is Alzheimer's disease and it affects some 400,000 people in Spain alone. However, no effective cure has been found till now.

Bionic Ear Technology Used for Gene Therapy may Help Restore Hearing Quality
New research suggests that the quality of hearing among people who have impaired hearing can be improved by using bionic ear technology.