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Whole gene replacement possible with new gene-editing tool

Researchers have developed a new tool that could potentially ‘cut’ out faulty genes and ‘paste’ in full-length copies of corrected […]

New AAV approach allows gene therapies to cross the blood-brain barrier

Written by Aaron Khemchandani, Science Writer. A new study, published in the journal Nature Biomedical Engineering, has shown how delivery […]

Single sex mice litters created using gene editing

Researchers at the Francis Crick Institute and the University of Kent have used gene-editing technology to create female-only and male-only […]

The rollercoaster ride of gene therapy

Where there is a symptom there is hopefully a treatment. For example, if I have a headache, I reach straight […]

Culturing ovarian follicles to foster in vitro gametogenesis

A recent study has demonstrated that the generation of functional ovarian follicle structures from mouse pluripotent stem cells in culture […]

Blind patient partially recovers after optogenetic therapy

French firm GenSight Biologics has published pioneering results showing the partial recovery of the first recipient that received their optogenetic […]

Gene therapy success in children with rare immunodeficiency

An investigational gene therapy for a rare, life-threatening inherited immunodeficiency disorder has been found to safely restore the immune systems […]

An Eye for an Eye: A New Optic Gene Therapy

A recent paper, published in Science, explored the use of optic gene therapy to treat Leber’s Hereditary Optic Neuropathy. Surprisingly, […]

Computational Genetics: A Massive Step for Neuroscience

Complex brain disorders are highly heritable and largely polygenic. They arise from a complex combination of risk genes, found in […]

Envisioning a CRISPR/Cas9-based therapy for retinitis pigmentosa

A proof-of-principle study has shown that template-free CRISPR/Cas9 gene editing can correct mutations that cause X-linked retinitis pigmentosa in mouse […]