Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice

Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice

Many inborn errors of metabolism require life-long treatments and, in severe conditions involving the liver, organ transplantation remains the only curative treatment. Non-integrative AAV-mediated gene therapy has shown efficacy in adult patients. However, treatment in pediatric or juvenile settings...

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Bibliographic Details
Main Authors: Michela Lisjak, Alessia De Caneva, Thibaut Marais, Elena Barbon, Maria Grazia Biferi, Fabiola Porro, Adi Barzel, Lorena Zentilin, Mark A. Kay, Federico Mingozzi, Andrés F. Muro
Format: Article
Language:English
Published: Frontiers Media S.A. 2022-03-01
Series:Frontiers in Genome Editing
Subjects:
GeneRide
albumin gene targeting
mouse model
coagulation factor IX
tail clip test
CRISPR/SaCas9
Online Access:https://www.frontiersin.org/articles/10.3389/fgeed.2022.785698/full

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https://www.frontiersin.org/articles/10.3389/fgeed.2022.785698/full

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