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Annals of Oncology : Official Journal of the European Society for Medical Oncology|July 19, 2013
A phase-III prevention trial of low-dose tamoxifen in postmenopausal hormone replacement therapy users: the HOT studyA DeCensi, B Bonanni, P Maisonneuve, et al.
Life Science Alliance|August 19, 2021
Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann-Pick disease type C1 miceCristin D Davidson, Alana L Gibson, Tansy Gu, et al.
Human Molecular Genetics|March 19, 2020
The vitamin B12 processing enzyme, mmachc, is essential for zebrafish survival, growth and retinal morphologyJennifer L Sloan, Nathan P Achilly, Madeline L Arnold, et al.
Molecular Metabolism|October 1, 2016
Defects in muscle branched-chain amino acid oxidation contribute to impaired lipid metabolismCarles Lerin, Allison B Goldfine, Tanner Boes, et al.
Molecular Therapy. Methods & Clinical Development|January 21, 2021
Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectorsRita Ferla, Marialuisa Alliegro, Margherita Dell'Anno, et al.
Human Gene Therapy|February 20, 2025
Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy ProgramRicha Madan Lomash, Jean Dehdashti, Oleg A Shchelochkov, et al.
Molecular Therapy. Methods & Clinical Development|September 6, 2021
ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemiaPetr O Ilyinskii, Alicia M Michaud, Gina L Rizzo, et al.
Human Gene Therapy|June 19, 2026
Adeno-Associated Virus Gene Therapy Translation: Lessons from Early Regulatory MeetingsRodica Stan, Richa Madan Lomash, Oleg A Shchelochkov, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|May 19, 2021
Severity modeling of propionic acidemia using clinical and laboratory biomarkersOleg A Shchelochkov, Irini Manoli, Paul Juneau, et al.
American Journal of Human Genetics|September 10, 2013
An X-linked cobalamin disorder caused by mutations in transcriptional coregulator HCFC1Hung-Chun Yu, Jennifer L Sloan, Gunter Scharer, et al.
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