Genome editing-based therapies typically aim to treat disease by correcting underlying genetic mutations in patient's cells.

A new method for safely inserting large chunks of DNA into genomes has now measured up in mice, potentially paving the way ...

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

This year’s Rare Disease Day event Feb. 27 at the Jacobs School of Medicine and Biomedical Sciences focused on updates in research and diagnostics, and advancements in treatment.

Cannabis and other illicit drugs are associated with higher stroke risk, underscoring the importance of addressing substance ...

You will be redirected to our submission process. Inborn errors of immunity (IEI), formerly known as primary immunodeficiencies, represent a heterogeneous group of more than 480 genetic disorders ...

Cambridge researchers map the convergence of biomarkers, digital phenotyping, and AI toward biologically grounded ...

"The stories are so outrageous," Senator Ron Johnson said. "It just appears that they’re looking for excuses to say no." ...

Hereditary ATTR-CM can run in families. Learn who may be at risk, when genetic testing is recommended, the benefits of screening, and how to talk with relatives.

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 ...

Large-scale evolutionary analysis shows most zoonotic viruses emerge without prior adaptation, while passing through a laboratory leaves detectable genetic signatures, offering a new tool to interpret ...

Yentli Soto Albrecht has a rare genetic form of ALS and is working on research at Penn that could one day prove life-saving.