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Molecular Therapy : the Journal of the American Society of Gene Therapy
|
February 1, 2024
Development of CNS tropic AAV1-like variants with reduced liver-targeting following systemic administration in mice
Matthieu Drouyer, Jessica Merjane, Deborah Nazareth, et al.
Nature
|
January 7, 2014
Selection and evaluation of clinically relevant AAV variants in a xenograft liver model
Leszek Lisowski, Allison P Dane, Kirk Chu, et al.
Human Gene Therapy
|
February 19, 2009
Transient in vivo beta-globin production after lentiviral gene transfer to hematopoietic stem cells in the nonhuman primate
Jun Hayakawa, Takahiro Ueda, Leszek Lisowski, et al.
ACS Nano
|
January 15, 2024
Neuronal Replenishment via Hydrogel-Rationed Delivery of Reprogramming Factors
Negar Mahmoudi, Yi Wang, Niamh Moriarty, et al.
Nature Biotechnology
|
December 27, 2005
A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference
Selda Samakoglu, Leszek Lisowski, Tulin Budak-Alpdogan, et al.
Nature Medicine
|
April 12, 2016
RNA interference-induced hepatotoxicity results from loss of the first synthesized isoform of microRNA-122 in mice
Paul N Valdmanis, Shuo Gu, Kirk Chu, et al.
Cells
|
April 30, 2021
Neurological Disorders Associated with WWOX Germline Mutations-A Comprehensive Overview
Ehud Banne, Baraa Abudiab, Sara Abu-Swai, et al.
Nature Communications
|
September 26, 2020
Engineering domain-inlaid SaCas9 adenine base editors with reduced RNA off-targets and increased on-target DNA editing
Minh Thuan Nguyen Tran, Mohd Khairul Nizam Mohd Khalid, Qi Wang, et al.
Molecular Therapy. Methods & Clinical Development
|
October 27, 2021
Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys
Julien Baruteau, Sharon C Cunningham, Berna Seker Yilmaz, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
July 1, 2020
In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation
Ana M Moreno, Xin Fu, Jie Zhu, et al.
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of 10
Search research articles
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Showing results (31-40 of 92) with videos related to
Sort By:
Page
of 10
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
February 1, 2024
Development of CNS tropic AAV1-like variants with reduced liver-targeting following systemic administration in mice
Matthieu Drouyer, Jessica Merjane, Deborah Nazareth, et al.
Nature
|
January 7, 2014
Selection and evaluation of clinically relevant AAV variants in a xenograft liver model
Leszek Lisowski, Allison P Dane, Kirk Chu, et al.
Human Gene Therapy
|
February 19, 2009
Transient in vivo beta-globin production after lentiviral gene transfer to hematopoietic stem cells in the nonhuman primate
Jun Hayakawa, Takahiro Ueda, Leszek Lisowski, et al.
ACS Nano
|
January 15, 2024
Neuronal Replenishment via Hydrogel-Rationed Delivery of Reprogramming Factors
Negar Mahmoudi, Yi Wang, Niamh Moriarty, et al.
Nature Biotechnology
|
December 27, 2005
A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference
Selda Samakoglu, Leszek Lisowski, Tulin Budak-Alpdogan, et al.
Nature Medicine
|
April 12, 2016
RNA interference-induced hepatotoxicity results from loss of the first synthesized isoform of microRNA-122 in mice
Paul N Valdmanis, Shuo Gu, Kirk Chu, et al.
Cells
|
April 30, 2021
Neurological Disorders Associated with WWOX Germline Mutations-A Comprehensive Overview
Ehud Banne, Baraa Abudiab, Sara Abu-Swai, et al.
Nature Communications
|
September 26, 2020
Engineering domain-inlaid SaCas9 adenine base editors with reduced RNA off-targets and increased on-target DNA editing
Minh Thuan Nguyen Tran, Mohd Khairul Nizam Mohd Khalid, Qi Wang, et al.
Molecular Therapy. Methods & Clinical Development
|
October 27, 2021
Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys
Julien Baruteau, Sharon C Cunningham, Berna Seker Yilmaz, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
July 1, 2020
In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation
Ana M Moreno, Xin Fu, Jie Zhu, et al.
Page
of 10