Cited in PMC for PubMed (Select 32363154) - PMC - NCBI

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Items: 12

Select item 107871521.

Combination HSCT and intravenous AAV-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy

Allison M. Bradbury, Jessica Bagel, Gary Swain, Keiko Miyadera, Jill P. Pesayco, Charles-Antoine Assenmacher, Becky Brisson, Ian Hendricks, Xiao H. Wang, Zachary Herbst, Nettie Pyne, Patricia Odonnell, G. Diane Shelton, Michael Gelb, Neil Hackett, Paul Szabolcs, Charles H. Vite, Maria Escolar

Mol Ther. 2024 Jan 3; 32(1): 44–58. Published online 2023 Nov 11. doi: 10.1016/j.ymthe.2023.11.014

PMCID:: PMC10787152

Currently embargoed: Free in PMC on Jan 3, 2025; PubMed

Select item 99641512.

Gene Therapy of Sphingolipid Metabolic Disorders

Alisa A. Shaimardanova, Valeriya V. Solovyeva, Shaza S. Issa, Albert A. Rizvanov

Int J Mol Sci. 2023 Feb; 24(4): 3627. Published online 2023 Feb 11. doi: 10.3390/ijms24043627

PMCID:: PMC9964151

Article PDF–755K

Select item 91427723.

Efficacy and Safety of a Krabbe Disease Gene Therapy

Juliette Hordeaux, Brianne A. Jeffrey, Jinlong Jian, Gourav R. Choudhury, Kristofer Michalson, Thomas W. Mitchell, Elizabeth L. Buza, Jessica Chichester, Cecilia Dyer, Jessica Bagel, Charles H. Vite, Allison M. Bradbury, James M. Wilson

Hum Gene Ther. May 2022; 33(9-10): 499–517. Published online 2022 May 16. doi: 10.1089/hum.2021.245

PMCID:: PMC9142772

Article PDF–1.6M

Select item 88025334.

Krabbe disease: New hope for an old disease

Allison M. Bradbury, Ernesto R. Bongarzone, Mark S. Sands

Neurosci Lett. Author manuscript; available in PMC 2022 Jan 31.

Published in final edited form as: Neurosci Lett. 2021 May 1; 752: 135841. Published online 2021 Mar 22. doi: 10.1016/j.neulet.2021.135841

PMCID:: PMC8802533

Article PDF–681K

Select item 87830825.

Krabbe disease: A personal perspective and hypothesis

Mohammad A. Rafi

Bioimpacts. 2022; 12(1): 3–7. Published online 2021 Dec 22. doi: 10.34172/bi.2021.23931

PMCID:: PMC8783082

Article PDF–518K

Select item 86338976.

Krabbe Disease: Prospects of Finding a Cure Using AAV Gene Therapy

Gibran Nasir, Rajiv Chopra, Fiona Elwood, Seemin S. Ahmed

Front Med (Lausanne) 2021; 8: 760236. Published online 2021 Nov 11. doi: 10.3389/fmed.2021.760236

PMCID:: PMC8633897

Article PDF–2.0M

Select item 85270177.

Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord

Thomas Leth Jensen, Casper René Gøtzsche, David P. D. Woldbye

Front Mol Neurosci. 2021; 14: 695937. Published online 2021 Oct 6. doi: 10.3389/fnmol.2021.695937

PMCID:: PMC8527017

Article PDF–686K

Select item 85022418.

Mechanisms of demyelination and neurodegeneration in globoid cell leukodystrophy

M. Laura Feltri, Nadav I. Weinstock, Jacob Favret, Narayan Dhimal, Lawrence Wrabetz, Daesung Shin

Glia. Author manuscript; available in PMC 2021 Oct 10.

Published in final edited form as: Glia. 2021 Oct; 69(10): 2309–2331. Published online 2021 Apr 14. doi: 10.1002/glia.24008

PMCID:: PMC8502241

Article PDF–1.3M

Select item 83960249.

Advances in the Diagnosis and Treatment of Krabbe Disease

David A Wenger, Paola Luzi, Mohammad A. Rafi

Int J Neonatal Screen. 2021 Sep; 7(3): 57. Published online 2021 Aug 18. doi: 10.3390/ijns7030057

PMCID:: PMC8396024

Article PDF–375K

Select item 825842110.

Emerging Concepts in Vector Development for Glial Gene Therapy: Implications for Leukodystrophies

Georg von Jonquieres, Caroline D. Rae, Gary D. Housley

Front Cell Neurosci. 2021; 15: 661857. Published online 2021 Jun 22. doi: 10.3389/fncel.2021.661857

PMCID:: PMC8258421

Article PDF–1.5M

Select item 802223211.

Can early treatment of twitcher mice with high dose AAVrh10-GALC eliminate the need for BMT?

Mohammad A Rafi, Paola Luzi, David A Wenger

Bioimpacts. 2021; 11(2): 135–146. Published online 2021 Feb 26. doi: 10.34172/bi.2021.21

PMCID:: PMC8022232

Article PDF–1.7M

Select item 780392012.

Achievements and beyond: Scientific trajectory of Professor Mohammad A. Rafi

Yadollah Omidi, Abass Alavi

Bioimpacts. 2021; 11(1): 1–4. Published online 2020 Dec 7. doi: 10.34172/bi.2021.01

PMCID:: PMC7803920

Article PDF–493K

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