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Article

AAV mediated genome engineering with a bypass coagulation factor alleviates the bleeding phenotype in a murine model of hemophilia B.

Thrombosis research - 1 Jun 2024

Sarangi Pratiksha, Kumar Narendra, Sambasivan Ramkumar, Ramalingam Sivaprakash, Amit Sonal, Chandra Dinesh, Jayandharan Giridhara R

Abstract excerpt

It is crucial to develop a long-term therapy that targets hemophilia A and B, including inhibitor-positive patients. We have developed an Adeno-associated virus (AAV) based strategy to integrate the bypass coagulation factor, activated FVII (murine, mFVIIa) gene into the Rosa26 locus using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 mediated gene-editing. AAV vectors designed for...

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Identifiers

PMID: 38718473
DOI: 10.1016/j.thromres.2024.04.031

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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