Liver Gene Therapy
Author:
Affiliation:
1. Department of Haematology, UCL-Cancer Institute, London, United Kingdom
2. Katharine Dormandy Haemophilia and Thrombosis Unit, Royal Free London NHS Foundation Trust, London, United Kingdom
3. Freeline Therapeutics, Stevenage, United Kingdom.
Publisher
Mary Ann Liebert Inc
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
https://www.liebertpub.com/doi/pdf/10.1089/hum.2022.169
Reference50 articles.
1. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer
2. Sustained Correction of X-Linked Severe Combined Immunodeficiency by ex Vivo Gene Therapy
3. A Serious Adverse Event after Successful Gene Therapy for X-Linked Severe Combined Immunodeficiency
4. LMO2 -Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1
5. Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria
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