AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria-Reference-Cited by-同舟云学术

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AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria

Published:2020-06 Issue: Volume:17 Page:234-245
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Richards Daelyn Y.,Winn Shelley R.,Dudley Sandra,Nygaard Sean,Mighell Taylor L.,Grompe Markus,Harding Cary O.

Funder

National PKU Alliance

National Institutes of Health

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference58 articles.

1. Genetics of phenylketonuria: then and now;Blau;Hum. Mutat.,2016

2. Phenylalanine hydroxylase: function, structure, and regulation;Flydal;IUBMB Life,2013

3. Living with phenylketonuria in adulthood: the PKU ATTITUDE study;Cazzorla;Mol. Genet. Metab. Rep.,2018

4. Molecular genetics and diagnosis of phenylketonuria: state of the art;Blau;Expert Rev. Mol. Diagn.,2014

5. Phenylketonuria: current treatments and future developments;Lichter-Konecki;Drugs,2019

Cited by 65 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Delivery of CRISPR/Cas9 system by AAV as vectors for gene therapy;Gene;2024-11

2. In vivo liver targeted genome editing as therapeutic approach: progresses and challenges;Frontiers in Genome Editing;2024-08-23

3. Gene editing in liver diseases;FEBS Letters;2024-07-30

4. A review on molecular scissoring with CRISPR/Cas9 genome editing technology;Toxicology Research;2024-07-01

5. Liver gene transfer for metabolite detoxification in inherited metabolic diseases;FEBS Letters;2024-06-17

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