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The Journal of Gene Medicine|March 26, 2018
Gene therapy clinical trials worldwide to 2017: An updateSamantha L Ginn, Anais K Amaya, Ian E Alexander, et al.
Molecular Biology of the Cell|November 29, 2023
Small-molecule correctors divert CFTR-F508del from ERAD by stabilizing sequential folding statesCeleste Riepe, Magda Wąchalska, Kirandeep K Deol, et al.
Biorxiv : the Preprint Server for Biology|September 25, 2023
Small molecule correctors divert CFTR-F508del from ERAD by stabilizing sequential folding statesCeleste Riepe, Magda Wąchalska, Kirandeep K Deol, et al.
Research Square|September 11, 2024
Secreted Particle Information Transfer (SPIT) - A Cellular Platform for In Vivo Genetic EngineeringCarsten T Charlesworth, Shota Homma, Fabian Suchy, et al.
Biorxiv : the Preprint Server for Biology|January 23, 2024
Secreted Particle Information Transfer (SPIT) - A Cellular Platform for In Vivo Genetic EngineeringCarsten T Charlesworth, Shota Homma, Fabian Suchy, et al.
JHEP Reports : Innovation in Hepatology|February 11, 2020
Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytesSamantha L Ginn, Anais K Amaya, Sophia H Y Liao, et al.
Molecular Therapy. Methods & Clinical Development|January 3, 2022
Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8Marti Cabanes-Creus, Renina Gale Navarro, Erhua Zhu, et al.
Molecular Therapy. Methods & Clinical Development|December 12, 2018
Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving FunctionalityMarti Cabanes-Creus, Samantha L Ginn, Anais K Amaya, et al.
Molecular Therapy. Methods & Clinical Development|March 25, 2025
Highly efficient in vivo hematopoietic stem cell transduction using an optimized self-complementary adeno-associated virusCarsten T Charlesworth, Shota Homma, Anais K Amaya, et al.
Nature Genetics|June 20, 2017
Identification of liver-specific enhancer-promoter activity in the 3' untranslated region of the wild-type AAV2 genomeGrant J Logan, Allison P Dane, Claus V Hallwirth, et al.
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