CRISPR Therapeutics has developed SyNTase editing, a proprietary, next-generation, site-specific gene correction platform.
Alpha-1 antitrypsin deficiency (AATD) is a recessive genetic disorder caused by single nucleotide variants (SNV) in the SERPINA1 gene encoding for alpha-1 antitrypsin (AAT), with the most common ...
Demonstrates modularity of Intellia’s in vivo liver insertion technology to durably restore protein, compared to traditional gene therapy Single-course administration of genome editing system provides ...
About Alpha-1 Antitrypsin Deficiency-Associated Liver Disease AATD is a rare genetic disorder associated with liver disease in children and adults and pulmonary disease in adults. AATD is estimated to ...
Please provide your email address to receive an email when new articles are posted on . WASHINGTON — Elevated Z polymer levels may predict lung and liver disease activity in patients with alpha 1 ...
About The Alpha-1 Project Mission statement: The Alpha-1 Project will work with patients, academia, pharmaceutical and biotech companies, and public health organizations in the relentless pursuit of ...
Tiny fat bubbles carrying gene therapy have successfully repaired DNA in the lungs and liver of animals with alpha-1 antitrypsin deficiency — a promising leap toward treating humans with this rare ...
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