A new genetic engineering strategy significantly reduces levels of tau in animal models of Alzheimer's disease. The treatment, which involves a single injection, appears to have long-last effects.
Researchers launch the first-in-human phase 1 clinical trial to assess the safety and efficacy of a gene therapy which will deliver BDNF to the brains of those with Alzheimer's and mild cognitive impairment.
Activating p38gamma, a naturally protective enzyme in the brain, may help to prevent the development of Alzheimer's disease symptoms. Researchers showed the naturally protective effects of p38gamma could be harnessed to improve memory in the later stages of Alzheimer's disease.
New gene therapy may help those with achromatopsia, or complete color blindness, to improve visual function. The treatment targets the CNGA3 gene, which is implicated in one-third of achromatopsia cases.
Zolgensma, an FDA approved gene therapy, supplements the production of the SMN protein to improve motor neuron function in children with spinal muscular atrophy after just one dose.
Study suggests new avenues for treating toxoplasmosis parasite infections.