University of Wisconsin–Madison researchers targeting a group of hereditary neurodegenerative diseases have found success ...

New data publishing 11/11 in Cell Stem Cell provides strong support for the first-in-human universal gene therapy trial for DBA through regulated GATA1 expression. Notably, it’s one of the first ...

Single-Dose Gene Therapy Is Potentially Life-Changing for ... holds a key to understanding how gene activity is encoded in the human genome. This breakthrough finding revealed a long-postulated ...

In recent years, scientists have created a range of new methods based on CRISPR-Cas technology for precisely editing the ...

Paxton Purdy's parents said he has an ultra-rare neurodegenerative genetic mutation. They hope they can raise enough money to ...

Explore the role and challenges of AAV characterization, the significance of capsid filling, and the best methods to enhance gene therapy.

Enrollment and dosing complete in single ascending dose clinical trial of anti-tau antibody VY7523 for Alzheimer’s; on track to generate ...

Its lead gene therapy candidate has the potential to be faster, less expensive, and more widely available than current CAR T ...

These pioneering companies are transforming delivery, medicine, and space access--potentially creating massive opportunities for early investors.

We recently published a list of 10 Most Promising Gene ... trials and human data. Morgan Stanley also noted that the Federal ...

University of Wisconsin-Madison researchers targeting a group of hereditary neurodegenerative diseases have found success ...