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Biorxiv : the Preprint Server for Biology
|
October 10, 2024
Expression-based selection identifies a microglia-tropic AAV capsid for direct and CSF routes of administration in mice
Miguel C Santoscoy, Paula Espinoza, Killian S Hanlon, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
November 20, 2024
CRISPR targeting of mmu-miR-21a through a single adeno-associated virus vector prolongs survival of glioblastoma-bearing mice
Lisa Nieland, Anne B Vrijmoet, Isabelle W Jetten, et al.
Molecular Therapy. Nucleic Acids
|
June 3, 2018
CRISPR/Cas9 Mediated Disruption of the Swedish APP Allele as a Therapeutic Approach for Early-Onset Alzheimer's Disease
Bence György, Camilla Lööv, Mikołaj P Zaborowski, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
January 14, 2017
Rescue of Hearing by Gene Delivery to Inner-Ear Hair Cells Using Exosome-Associated AAV
Bence György, Cyrille Sage, Artur A Indzhykulian, et al.
Molecular Therapy. Methods & Clinical Development
|
December 3, 2019
Selection of an Efficient AAV Vector for Robust CNS Transgene Expression
Killian S Hanlon, Jonah C Meltzer, Tetyana Buzhdygan, et al.
Biorxiv : the Preprint Server for Biology
|
January 30, 2023
Base editing as a genetic treatment for spinal muscular atrophy
Christiano R R Alves, Leillani L Ha, Rebecca Yaworski, et al.
Molecular Therapy. Methods & Clinical Development
|
July 18, 2022
Gene replacement therapy in a schwannoma mouse model of neurofibromatosis type 2
Shilpa Prabhakar, Roberta L Beauchamp, Pike See Cheah, et al.
Molecular Therapy. Methods & Clinical Development
|
May 15, 2024
Erratum: Gene replacement therapy in a schwannoma mouse model of neurofibromatosis type 2
Shilpa Prabhakar, Roberta L Beauchamp, Pike See Cheah, et al.
Blood Advances
|
January 4, 2018
Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors
Amine Meliani, Florence Boisgerault, Zachary Fitzpatrick, et al.
Nature Biomedical Engineering
|
December 6, 2023
Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy
Christiano R R Alves, Leillani L Ha, Rebecca Yaworski, et al.
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Search research articles
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Showing results (51-60 of 74) with videos related to
Sort By:
Page
of 8
Biorxiv : the Preprint Server for Biology
|
October 10, 2024
Expression-based selection identifies a microglia-tropic AAV capsid for direct and CSF routes of administration in mice
Miguel C Santoscoy, Paula Espinoza, Killian S Hanlon, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
November 20, 2024
CRISPR targeting of mmu-miR-21a through a single adeno-associated virus vector prolongs survival of glioblastoma-bearing mice
Lisa Nieland, Anne B Vrijmoet, Isabelle W Jetten, et al.
Molecular Therapy. Nucleic Acids
|
June 3, 2018
CRISPR/Cas9 Mediated Disruption of the Swedish APP Allele as a Therapeutic Approach for Early-Onset Alzheimer's Disease
Bence György, Camilla Lööv, Mikołaj P Zaborowski, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
January 14, 2017
Rescue of Hearing by Gene Delivery to Inner-Ear Hair Cells Using Exosome-Associated AAV
Bence György, Cyrille Sage, Artur A Indzhykulian, et al.
Molecular Therapy. Methods & Clinical Development
|
December 3, 2019
Selection of an Efficient AAV Vector for Robust CNS Transgene Expression
Killian S Hanlon, Jonah C Meltzer, Tetyana Buzhdygan, et al.
Biorxiv : the Preprint Server for Biology
|
January 30, 2023
Base editing as a genetic treatment for spinal muscular atrophy
Christiano R R Alves, Leillani L Ha, Rebecca Yaworski, et al.
Molecular Therapy. Methods & Clinical Development
|
July 18, 2022
Gene replacement therapy in a schwannoma mouse model of neurofibromatosis type 2
Shilpa Prabhakar, Roberta L Beauchamp, Pike See Cheah, et al.
Molecular Therapy. Methods & Clinical Development
|
May 15, 2024
Erratum: Gene replacement therapy in a schwannoma mouse model of neurofibromatosis type 2
Shilpa Prabhakar, Roberta L Beauchamp, Pike See Cheah, et al.
Blood Advances
|
January 4, 2018
Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors
Amine Meliani, Florence Boisgerault, Zachary Fitzpatrick, et al.
Nature Biomedical Engineering
|
December 6, 2023
Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy
Christiano R R Alves, Leillani L Ha, Rebecca Yaworski, et al.
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