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» NGGT-002 gene therapy shows potential for treating phenylketonuria
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Endocrine/Metabolic
NGGT-002 gene therapy shows potential for treating phenylketonuria
May 29, 2023
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NGGT (Suzhou) Biotechnology Co. Ltd. has presented preclinical data on an AAV vector approach that expresses human PAH, rAAV8-PAH, also known as NGGT-002. NGGT-002 has liver tropism and it was codon-optimized for expressing PAH in the liver.
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