A new study published in Neurology, led by Queen Mary University of London, has revealed that people of South Asian, African ...

In an exciting scientific first, researchers at the Allen Institute successfully designed a new gene therapy that reversed symptoms related to SYNGAP1-related disorders (SRD) in mice. These are a ...

Genetic defects are exceedingly common, which is not surprising considering just how many cells make up our bodies, including our reproductive cells. While most of these defects have no or only minor ...

Bayer has discontinued an early-stage clinical gene therapy for a rare genetic disorder in favor of a similar candidate. | Bayer has discontinued an early-stage clinical gene therapy for a rare ...

A new federal proposal aimed at speeding approval of gene therapies for ultra-rare diseases could offer renewed hope to North Texans like Kasey and ...

This Ice Age pair of hunter-gatherers were both shorter than average for their population – one of them markedly so – and now we finally know why. A team of researchers from Europe re-examined the ...

Genetic diseases are notoriously challenging to treat, especially when each condition requires a tailored approach. With over 10,000 known genetic disorders, developing individual therapies for each ...

A closer examination of the APOL1 gene in Black patients with kidney disease can provide more accurate diagnoses than current ...

An integrin mutation impairs skin T cell migration, enabling HPV proliferation that causes warts and lesions in a rare genetic dermatological condition.

The US FDA approved leucovorin only for a rare genetic cerebral folate deficiency affecting about 1 in a million people, rejecting earlier White House suggestions of its broader use for autism ...

Chronic kidney disease (CKD) is a public health challenge that affects more than 800 million people worldwide. 1 CKD can be caused by a variety of disease processes. Many causes are difficult to ...