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1.
Systemic AAV8-mediated delivery of a functional copy of muscle glycogen phosphorylase (Pygm) ameliorates disease in a murine model of McArdle disease.
Hum Mol Genet
; 29(1): 20-30, 2020 01 01.
Artículo
en Inglés
| MEDLINE | ID: mdl-31511858
2.
Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors.
Hum Gene Ther
; 34(7-8): 273-288, 2023 04.
Artículo
en Inglés
| MEDLINE | ID: mdl-36927149
3.
Engineering domain-inlaid SaCas9 adenine base editors with reduced RNA off-targets and increased on-target DNA editing.
Nat Commun
; 11(1): 4871, 2020 09 25.
Artículo
en Inglés
| MEDLINE | ID: mdl-32978399
4.
Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes.
JHEP Rep
; 2(1): 100065, 2020 Feb.
Artículo
en Inglés
| MEDLINE | ID: mdl-32039406
5.
Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2.
Mol Ther Methods Clin Dev
; 17: 1139-1154, 2020 Jun 12.
Artículo
en Inglés
| MEDLINE | ID: mdl-32490035
6.
Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality.
Mol Ther Methods Clin Dev
; 12: 71-84, 2019 Mar 15.
Artículo
en Inglés
| MEDLINE | ID: mdl-30534580
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