Gene therapy may be 'one-shot stop' for rare bone disease
For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...
technologynetworks11h
Gene Therapy Shows Promise for Rare Bone Disease in Mice
Researchers have developed a gene therapy that may provide a long-term treatment for hypophosphatasia. Unlike current ...
Hosted on MSN1mon
Discovery of enzyme pathway may lead to lifesaving leishmaniasis treatments
Leishmaniasis, spread by a sandfly vector in warmer climates ... known as the CYP5122A1 enzyme. Therefore, azole antifungals targeting the CYP5122A1 enzyme as well as the traditional CYP51 ...
GEN20h
AAV Delivered NanoCas CRISPR System Edits Muscle in Non-Human Primates
Potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector.
Mammoth Biosciences Announces New Results on NanoCas – the First Efficient Ultracompact Extrahepatic Gene Editor
NanoCas, an ultracompact nuclease, enables efficient muscle editing in NHPs using a single AAV - advancing gene editing for hard-to-target tissues.
Business Insider12d
Genethon and Eukarÿs announce a strategic partnership to develop a breakthrough technology to reduce the biomanufacturing cost of gene therapies
Eukarÿs has developed an engineered enzyme, C3P3, which significantly increases the production ... vectors, one of the main vector types used to deliver therapeutic genes to patients. As part of this ...
pharmaphorum11d
Regenxbio expands MPS I gene therapy trial after first look at data
The results come from five patients enrolled in the biotech's ingoing phase 1/2 study of RGX-111 – which uses an adeno-associated viral vector (AAV9) to deliver a gene coding for an enzyme that ...
precisionmedicineonline19h
UniQure Testing Higher Gene Therapy Dose in Phase I/II Fabry Disease Trial
The firm will begin testing a second dose cohort with AMT-191 after receiving a positive recommendation from a data monitoring committee.