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Showing results (181-190 of 194) with videos related to

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Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology|January 24, 1998
Intensive chemotherapy and bone marrow rescue for young children with newly diagnosed malignant brain tumorsW P Mason, A Grovas, S Halpern, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 28, 2021
Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe diseaseGregory J Heller, Michael S Marshall, Yazan Issa, et al.
Acta Neuropathologica Communications|July 23, 2025
Gene therapy ameliorates neuromuscular pathology in CLN3 diseaseEwa A Ziółkowska, Albina Jablonka-Shariff, Letitia L Williams, et al.
Scientific Reports|July 15, 2021
Substrate reduction therapy for Krabbe disease and metachromatic leukodystrophy using a novel ceramide galactosyltransferase inhibitorMichael C Babcock, Christina R Mikulka, Bing Wang, et al.
Proceedings of the National Academy of Sciences of the United States of America|September 19, 2019
Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic targetYedda Li, Yue Xu, Bruno A Benitez, et al.
Biorxiv : the Preprint Server for Biology|November 28, 2024
Haploinsufficiency of lysosomal enzyme genes in Alzheimer's diseaseBruno A Benitez, Clare E Wallace, Maulikkumar Patel, et al.
Biorxiv : the Preprint Server for Biology|February 20, 2025
Identifying and treating CLN3 disease outside the central nervous systemEwa A Ziółkowska, Albina Jablonka-Shariff, Letitia L Williams, et al.
The Journal of Clinical Investigation|August 11, 2020
Krabbe disease successfully treated via monotherapy of intrathecal gene therapyAllison M Bradbury, Jessica H Bagel, Duc Nguyen, et al.
Nature Communications|June 13, 2023
Embryonic vitamin D deficiency programs hematopoietic stem cells to induce type 2 diabetesJisu Oh, Amy E Riek, Kevin T Bauerle, et al.
Cell Stem Cell|May 1, 2012
Background mutations in parental cells account for most of the genetic heterogeneity of induced pluripotent stem cellsMargaret A Young, David E Larson, Chiao-Wang Sun, et al.
Pageof 20

Showing results (181-190 of 194) with videos related to

Sort By:
Pageof 20
Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology|January 24, 1998
Intensive chemotherapy and bone marrow rescue for young children with newly diagnosed malignant brain tumorsW P Mason, A Grovas, S Halpern, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 28, 2021
Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe diseaseGregory J Heller, Michael S Marshall, Yazan Issa, et al.
Acta Neuropathologica Communications|July 23, 2025
Gene therapy ameliorates neuromuscular pathology in CLN3 diseaseEwa A Ziółkowska, Albina Jablonka-Shariff, Letitia L Williams, et al.
Scientific Reports|July 15, 2021
Substrate reduction therapy for Krabbe disease and metachromatic leukodystrophy using a novel ceramide galactosyltransferase inhibitorMichael C Babcock, Christina R Mikulka, Bing Wang, et al.
Proceedings of the National Academy of Sciences of the United States of America|September 19, 2019
Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic targetYedda Li, Yue Xu, Bruno A Benitez, et al.
Biorxiv : the Preprint Server for Biology|November 28, 2024
Haploinsufficiency of lysosomal enzyme genes in Alzheimer's diseaseBruno A Benitez, Clare E Wallace, Maulikkumar Patel, et al.
Biorxiv : the Preprint Server for Biology|February 20, 2025
Identifying and treating CLN3 disease outside the central nervous systemEwa A Ziółkowska, Albina Jablonka-Shariff, Letitia L Williams, et al.
The Journal of Clinical Investigation|August 11, 2020
Krabbe disease successfully treated via monotherapy of intrathecal gene therapyAllison M Bradbury, Jessica H Bagel, Duc Nguyen, et al.
Nature Communications|June 13, 2023
Embryonic vitamin D deficiency programs hematopoietic stem cells to induce type 2 diabetesJisu Oh, Amy E Riek, Kevin T Bauerle, et al.
Cell Stem Cell|May 1, 2012
Background mutations in parental cells account for most of the genetic heterogeneity of induced pluripotent stem cellsMargaret A Young, David E Larson, Chiao-Wang Sun, et al.
Pageof 20