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Human Gene Therapy
|
February 20, 2025
Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy Program
Richa 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 acidemia
Petr 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 Meetings
Rodica 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 biomarkers
Oleg 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 HCFC1
Hung-Chun Yu, Jennifer L Sloan, Gunter Scharer, et al.
American Journal of Medical Genetics. Part A
|
February 2, 2022
Probing the functional consequence and clinical relevance of CD320 p.E88del, a variant in the transcobalamin receptor gene
Faith Pangilinan, David Watkins, David Bernard, et al.
Nature Genetics
|
March 3, 2015
Germline gain-of-function mutations in AFF4 cause a developmental syndrome functionally linking the super elongation complex and cohesin
Kosuke Izumi, Ryuichiro Nakato, Zhe Zhang, et al.
Brain : a Journal of Neurology
|
February 1, 2022
ZBTB11 dysfunction: spectrum of brain abnormalities, biochemical signature and cellular consequences
Dulika Sumathipala, Petter Strømme, Zohreh Fattahi, et al.
JCI Insight
|
September 9, 2025
Dissecting the effect of mitochondrial BCAT inhibition in methylmalonic acidemia
Madeline G Hemmingsen, Guo-Fang Zhang, Yunhan Ma, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
May 11, 2021
Correction to: 1-<sup>13</sup>C-propionate breath testing as a surrogate endpoint to assess efficacy of liver-directed therapies in methylmalonic acidemia (MMA)
Irini Manoli, Alexandra R Pass, Elizabeth A Harrington, et al.
Page
of 18
Search research articles
Search
Showing results (161-170 of 178) with videos related to
Sort By:
Page
of 18
Human Gene Therapy
|
February 20, 2025
Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy Program
Richa 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 acidemia
Petr 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 Meetings
Rodica 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 biomarkers
Oleg 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 HCFC1
Hung-Chun Yu, Jennifer L Sloan, Gunter Scharer, et al.
American Journal of Medical Genetics. Part A
|
February 2, 2022
Probing the functional consequence and clinical relevance of CD320 p.E88del, a variant in the transcobalamin receptor gene
Faith Pangilinan, David Watkins, David Bernard, et al.
Nature Genetics
|
March 3, 2015
Germline gain-of-function mutations in AFF4 cause a developmental syndrome functionally linking the super elongation complex and cohesin
Kosuke Izumi, Ryuichiro Nakato, Zhe Zhang, et al.
Brain : a Journal of Neurology
|
February 1, 2022
ZBTB11 dysfunction: spectrum of brain abnormalities, biochemical signature and cellular consequences
Dulika Sumathipala, Petter Strømme, Zohreh Fattahi, et al.
JCI Insight
|
September 9, 2025
Dissecting the effect of mitochondrial BCAT inhibition in methylmalonic acidemia
Madeline G Hemmingsen, Guo-Fang Zhang, Yunhan Ma, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
May 11, 2021
Correction to: 1-<sup>13</sup>C-propionate breath testing as a surrogate endpoint to assess efficacy of liver-directed therapies in methylmalonic acidemia (MMA)
Irini Manoli, Alexandra R Pass, Elizabeth A Harrington, et al.
Page
of 18