Human Gene Therapy
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5371
(FIVE YEARS 493)

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143
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Published By Mary Ann Liebert

1557-7422, 1043-0342
Latest Documents Most Cited Documents Contributed Authors Related Sources Related Keywords

Efficacy and Safety of FX201, a Novel Intra-Articular IL-1Ra Gene Therapy for Osteoarthritis Treatment, in a Rat Model

2021 ◽  
Author(s):  
Rebecca Senter ◽  
Rogely Boyce ◽  
Marko Repic ◽  
Emily Walsh Martin ◽  
Monika Chabicovsky ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Rat Model ◽  
Efficacy And Safety ◽  
Osteoarthritis Treatment

Strategies for optimization of the CRISPR-based genome editing system for enhanced editing specificity

2021 ◽  
Author(s):  
Yangmin Wang ◽  
Haozheng Wang ◽  
Yingzhen Jian ◽  
Zhongtao Luo ◽  
Hongwei Shao ◽  
...  
Keyword(s):  
Genome Editing

Insight & Development of Advanced rAAV Analysis Tools Exploiting Single Particle Quantification by Multidimensional ddPCR.

2021 ◽  
Author(s):  
Jeanette Zanker ◽  
Sara Lázaro-Petri ◽  
Daniela Hüser ◽  
Regine Heilbronn ◽  
Adrien Savy
Keyword(s):  
Single Particle ◽  
Analysis Tools ◽  
Particle Quantification

scholarly journals Biodistribution and Tolerability of AAV-PHP.B-CBh-SMN1 in Wistar Han Rats and Cynomolgus Macaques Reveal Different Toxicologic Profiles

2021 ◽  
Author(s):  
Xavier PALAZZI ◽  
Ingrid Pardo ◽  
Madhu Sirivelu ◽  
Leah Newman ◽  
Steven Kumpf ◽  
...  
Keyword(s):  
Cynomolgus Macaques

Prevalence of pre-existing neutralizing antibodies against AAV serotypes 1, 2, 5, 6, 8, and 9 in sera of different pig strains

2021 ◽  
Author(s):  
Yanshan Dai ◽  
Uma Kavita ◽  
Margit H Lampen ◽  
Sander Gielen ◽  
Glen Banks ◽  
...  
Keyword(s):  
Neutralizing Antibodies
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