Molecular Therapy — Methods & Clinical Development
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scholarly journals A universal strategy for AAV delivery of base editors to correct genetic point mutations in neonatal PKU mice

2022 ◽  
Author(s):  
Lifang Zhou ◽  
Jing Su ◽  
Jie Long ◽  
Rui Tao ◽  
Wenling Tang ◽  
...  
Keyword(s):  
Point Mutations

scholarly journals Delivery of nVEGFi using AAV8 for the treatment of neovascular age-related macular degeneration

2022 ◽  
Author(s):  
Kaiqin She ◽  
Jing Su ◽  
Qingnan Wang ◽  
Yi Liu ◽  
Xiaomei Zhong ◽  
...  
Keyword(s):  
Macular Degeneration ◽  
Age Related Macular Degeneration ◽  
Age Related

scholarly journals Confirmatory Detection of Neutralizing Antibodies to AAV Gene Therapy Using a Cell-based Transduction Inhibition Assay

2022 ◽  
Author(s):  
Theresa Kasprzyk ◽  
Sabrina Triffault ◽  
Brian Long ◽  
Stephen J. Zoog ◽  
Christian Vettermann
Keyword(s):  
Gene Therapy ◽  
Neutralizing Antibodies ◽  
Inhibition Assay ◽  
A Cell

scholarly journals Consensus guidelines for the validation of quantitative RT-qPCR assays in clinical research by the CardioRNA consortium

2022 ◽  
Author(s):  
David de Gonzalo-Calvo ◽  
Monica Marchese ◽  
Jan Hellemans ◽  
Fay Betsou ◽  
Nanna Lond Skov Frisk ◽  
...  
Keyword(s):  
Clinical Research ◽  
Consensus Guidelines

scholarly journals High concordance of ELISA and neutralization assays allows for the detection of antibodies to individual AAV serotypes

2022 ◽  
Author(s):  
Matthew R. Gardner ◽  
Desiree E. Mendes ◽  
Claudia P. Muniz ◽  
José M. Martinez-Navio ◽  
Sebastian P. Fuchs ◽  
...  
Keyword(s):  
High Concordance

scholarly journals Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette

2021 ◽  
Vol 23 ◽  
pp. 98-107
Author(s):  
Sandeep R.P. Kumar ◽  
Jun Xie ◽  
Shilang Hu ◽  
Jihye Ko ◽  
Qifeng Huang ◽  
...  
Keyword(s):  
Gene Transfer ◽  
Coagulation Factor ◽  
Factor Ix ◽  
Expression Cassette ◽  
Coagulation Factor Ix

scholarly journals Immunological evaluation of an inactivated SARS-CoV-2 vaccine in rhesus macaques

2021 ◽  
Vol 23 ◽  
pp. 108-118
Author(s):  
Hongbo Chen ◽  
Zhongping Xie ◽  
Runxiang Long ◽  
Shengtao Fan ◽  
Heng Li ◽  
...  
Keyword(s):  
Rhesus Macaques ◽  
Immunological Evaluation

scholarly journals Accelerating clinical scale production of BCMA CAR T cells with defined maturation stages

2021 ◽  
Author(s):  
Jara J. Joedicke ◽  
Ulrich Großkinsky ◽  
Kerstin Gerlach ◽  
Annette Künkele ◽  
Uta E. Höpken ◽  
...  
Keyword(s):  
T Cells ◽  
Scale Production ◽  
Clinical Scale ◽  
Car T Cells ◽  
Car T ◽  
Maturation Stages

scholarly journals Voluntary wheel running complements microdystrophin gene therapy to improve muscle function in mdx mice

2021 ◽  
Vol 23 ◽  
pp. 460
Author(s):  
Shelby E. Hamm ◽  
Daniel D. Fathalikhani ◽  
Katherine E. Bukovec ◽  
Adele K. Addington ◽  
Haiyan Zhang ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Muscle Function ◽  
Wheel Running ◽  
Mdx Mice ◽  
Voluntary Wheel Running ◽  
Voluntary Wheel

scholarly journals Enzymatically produced piggyBac transposon vectors for efficient non-viral manufacturing of CD19-specific CAR T cells

2021 ◽  
Vol 23 ◽  
pp. 119-127
Author(s):  
Iva Kaštánková ◽  
Martin Štach ◽  
Hana Žižková ◽  
Pavlína Ptáčková ◽  
Kristýna Šmilauerová ◽  
...  
Keyword(s):  
T Cells ◽  
Piggybac Transposon ◽  
Car T Cells ◽  
Car T
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