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Molecular Therapy : the Journal of the American Society of Gene Therapy
|
October 26, 2024
Quantifying and mitigating motor phenotypes induced by antisense oligonucleotides in the central nervous system
Michael P Moazami, Julia M Rembetsy-Brown, Samantha L Sarli, et al.
Nature Medicine
|
December 24, 2021
Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide
Hélène Tran, Michael P Moazami, Huiya Yang, et al.
Elife
|
October 19, 2021
5'-Modifications improve potency and efficacy of DNA donors for precision genome editing
Krishna S Ghanta, Zexiang Chen, Aamir Mir, et al.
Nucleic Acids Research
|
August 3, 2022
Intratracheally administered LNA gapmer antisense oligonucleotides induce robust gene silencing in mouse lung fibroblasts
Minwook Shin, Io Long Chan, Yuming Cao, et al.
Nature Biotechnology
|
September 25, 2024
Increasing intracellular dNTP levels improves prime editing efficiency
Pengpeng Liu, Karthikeyan Ponnienselvan, Thomas Nyalile, et al.
Biorxiv : the Preprint Server for Biology
|
June 5, 2026
Fully Modified SpyCas9 Guide RNAs Enable Robust Genome Editing In Cells and In Vivo
Kim Anh Vu, Han Zhang, Nadia Amrani, et al.
European Urology Oncology
|
January 23, 2024
Oncologic Outcomes in Patients with Residual Upper Tract Urothelial Carcinoma Following Neoadjuvant Chemotherapy
Sean A Fletcher, Maximilian Pallauf, Emelia K Watts, et al.
Lancet (London, England)
|
May 25, 2025
Antisense oligonucleotide jacifusen for FUS-ALS: an investigator-initiated, multicentre, open-label case series
Neil A Shneider, Matthew B Harms, Vlad A Korobeynikov, et al.
Nature Neuroscience
|
November 1, 2025
TDP-43-dependent mis-splicing of KCNQ2 triggers intrinsic neuronal hyperexcitability in ALS/FTD
Brian J Joseph, Kelly A Marshall, Peter Harley, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
March 9, 2023
Divalent siRNAs are bioavailable in the lung and efficiently block SARS-CoV-2 infection
Vignesh N Hariharan, Minwook Shin, Ching-Wen Chang, et al.
Page
of 19
Search research articles
Search
Showing results (171-180 of 184) with videos related to
Sort By:
Page
of 19
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
October 26, 2024
Quantifying and mitigating motor phenotypes induced by antisense oligonucleotides in the central nervous system
Michael P Moazami, Julia M Rembetsy-Brown, Samantha L Sarli, et al.
Nature Medicine
|
December 24, 2021
Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide
Hélène Tran, Michael P Moazami, Huiya Yang, et al.
Elife
|
October 19, 2021
5'-Modifications improve potency and efficacy of DNA donors for precision genome editing
Krishna S Ghanta, Zexiang Chen, Aamir Mir, et al.
Nucleic Acids Research
|
August 3, 2022
Intratracheally administered LNA gapmer antisense oligonucleotides induce robust gene silencing in mouse lung fibroblasts
Minwook Shin, Io Long Chan, Yuming Cao, et al.
Nature Biotechnology
|
September 25, 2024
Increasing intracellular dNTP levels improves prime editing efficiency
Pengpeng Liu, Karthikeyan Ponnienselvan, Thomas Nyalile, et al.
Biorxiv : the Preprint Server for Biology
|
June 5, 2026
Fully Modified SpyCas9 Guide RNAs Enable Robust Genome Editing In Cells and In Vivo
Kim Anh Vu, Han Zhang, Nadia Amrani, et al.
European Urology Oncology
|
January 23, 2024
Oncologic Outcomes in Patients with Residual Upper Tract Urothelial Carcinoma Following Neoadjuvant Chemotherapy
Sean A Fletcher, Maximilian Pallauf, Emelia K Watts, et al.
Lancet (London, England)
|
May 25, 2025
Antisense oligonucleotide jacifusen for FUS-ALS: an investigator-initiated, multicentre, open-label case series
Neil A Shneider, Matthew B Harms, Vlad A Korobeynikov, et al.
Nature Neuroscience
|
November 1, 2025
TDP-43-dependent mis-splicing of KCNQ2 triggers intrinsic neuronal hyperexcitability in ALS/FTD
Brian J Joseph, Kelly A Marshall, Peter Harley, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
March 9, 2023
Divalent siRNAs are bioavailable in the lung and efficiently block SARS-CoV-2 infection
Vignesh N Hariharan, Minwook Shin, Ching-Wen Chang, et al.
Page
of 19