If scientists can find the genetic basis for the axolotl’s ability to regenerate, they might be able to find ways to restore damaged tissue in humans. But they have been thwarted in the attempt by another peculiarity of the axolotl — it has the largest genome of any animal yet sequenced, 10 times larger than that of humans
New gene correction therapy for Duchenne muscular dystrophy
Proof-of-concept shows genes implicated in Alzheimer’s disease can be accurately edited with 90% efficiency in human stem cells
Collaborative effort by investing in the development of curative therapies that can be delivered safely, effectively and affordably in low-resource settings
Research team finds that a nontoxic molecule closely related to resveratrol can overcome barriers to delivering gene therapy into stem cells
Structures are alternatives to viral vectors for gene therapy using the CRISPR-Cas9 system
Duchenne muscular dystrophy (DMD) is a rare but devastating genetic disorder that causes muscle loss and physical impairments. Researchers have shown that CRISPR may provide the means for lifelong correction of the genetic mutation responsible for the disorder
Researchers have unravelled parts of a mechanism that may one day help to treat Rett syndrome and other genetic disorders linked to the X chromosome.
A new gene therapy turns glial cells into neurons, repairing damage that results from stroke and significantly improving motor function
