Rodent model for serpina1 gene correction

TW202635202APending Publication Date: 2026-09-01GENZYME CORP
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Patent Information

Application Number
TW114138430
Authority / Receiving Office
TW · TW
Patent Type
Applications
Current Assignee / Owner
Priority Date
2024-12-05
Filing Date
2025-10-03
Publication Date
2026-09-01

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Abstract

Provided herein are humanized rodent models comprising a human SERPINA1 transgene, optionally wherein the rodents develop Alpha-1 antitrypsin deficiency (A1ATD). Further provided herein are methods related to the use of the humanized rodent models for screening therapeutic efficacy for A1ATD, for example, for screening the efficacy of any treatment selected from the group consisting of liver transplantation, respiratory agents, purified alpha-1 antitrypsin (A1AT) protein, and gene therapy.
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