Results 231 to 240 of about 121,774 (253)

AAV

2013
openaire   +1 more source

Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B

Molecular Therapy, 2020
Lindsey A George, Margaret Ragni, John E J Rasko   +2 more
exaly  

AAV Puzzle

JCR Journal of Clinical Rheumatology, 2015
openaire   +1 more source

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

Molecular Therapy - Methods and Clinical Development, 2018
Pasqualina Colella, Giuseppe Ronzitti, Federico Mingozzi   +2 more
exaly  

AAV-ID: A Rapid and Robust Assay for Batch-to-Batch Consistency Evaluation of AAV Preparations

Molecular Therapy, 2017
Eva Andres-Mateos, Anna Maurer, Magalie Penaud-Budloo   +2 more
exaly  

AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer

Molecular Therapy, 2020
Helena Costa-Verdera, Federico Mingozzi
exaly  

The Effect of CpG Sequences on Capsid-Specific CD8+ T Cell Responses to AAV Vector Gene Transfer

Molecular Therapy, 2020
Yan Li, Federico Mingozzi, Hildegund C J Ertl   +2 more
exaly  

AAV Plasmids

Cold Spring Harbor Protocols, 2015
openaire   +1 more source

Effective delivery of large genes to the retina by dual AAV vectors

EMBO Molecular Medicine, 2014
Ivana Trapani, Pasqualina Colella, Massimo Giunti   +2 more
exaly  

Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity

Molecular Therapy, 2018
Katja Pekrun, Erhua Zhu, Sean Nygaard
exaly