TUCSON, Ariz., August 6, 2024 — Critical Path Institute’s (C-Path) Critical Path for Alpha-1 Antitrypsin Deficiency (CPA-1) consortium today announced the addition of several key advisory members. The ...
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 ...
BEAM-302 demonstrated safety and efficacy in increasing AAT levels and reducing mutant protein in alpha-1 antitrypsin deficiency patients. Beam Therapeutics announced promising initial results from ...
Alpha-1 antitrypsin (AAT) deficiency is a genetic disease where your body doesn’t make enough normal functioning AAT. AAT is a protein your liver makes. AAT has many important roles in your body, ...
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Rescued by fat bubbles: UTSW scientists treat rare genetic disease with designer molecule
DALLAS – 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 ...
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