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C Frank Bennett

Showing results (141-150 of 169) with videos related to

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The New England Journal of Medicine|November 2, 2017
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular AtrophyRichard S Finkel, Eugenio Mercuri, Basil T Darras, et al.
Science Translational Medicine|October 5, 2018
Huntingtin suppression restores cognitive function in a mouse model of Huntington's diseaseAmber L Southwell, Holly B Kordasiewicz, Douglas Langbehn, et al.
Biorxiv : the Preprint Server for Biology|November 24, 2025
A splice-switching antisense oligonucleotide approach for pediatric genetic epilepsiesHaley B Dame, Hema Kopalle, Belen Miñana, et al.
Neuron|October 22, 2013
RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense interventionChristopher J Donnelly, Ping-Wu Zhang, Jacqueline T Pham, et al.
Science Translational Medicine|May 14, 2025
Intra-amniotic antisense oligonucleotide treatment improves phenotypes in preclinical models of spinal muscular atrophyBeltran Borges, Stephen M Brown, Wan-Jin Chen, et al.
Nature Neuroscience|October 2, 2012
Divergent roles of ALS-linked proteins FUS/TLS and TDP-43 intersect in processing long pre-mRNAsClotilde Lagier-Tourenne, Magdalini Polymenidou, Kasey R Hutt, et al.
The New England Journal of Medicine|February 15, 2018
Nusinersen versus Sham Control in Later-Onset Spinal Muscular AtrophyEugenio Mercuri, Basil T Darras, Claudia A Chiriboga, et al.
The Journal of Clinical Investigation|October 8, 2019
Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatmentDaniel M Ramos, Constantin d'Ydewalle, Vijayalakshmi Gabbeta, et al.
The Journal of Clinical Investigation|July 17, 2018
Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS modelsAlex McCampbell, Tracy Cole, Amy J Wegener, et al.
Nucleic Acids Research|May 12, 2019
Conjugation of hydrophobic moieties enhances potency of antisense oligonucleotides in the muscle of rodents and non-human primatesMichael E Østergaard, Michaela Jackson, Audrey Low, et al.
Pageof 17

Showing results (141-150 of 169) with videos related to

Sort By:
Pageof 17
The New England Journal of Medicine|November 2, 2017
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular AtrophyRichard S Finkel, Eugenio Mercuri, Basil T Darras, et al.
Science Translational Medicine|October 5, 2018
Huntingtin suppression restores cognitive function in a mouse model of Huntington's diseaseAmber L Southwell, Holly B Kordasiewicz, Douglas Langbehn, et al.
Biorxiv : the Preprint Server for Biology|November 24, 2025
A splice-switching antisense oligonucleotide approach for pediatric genetic epilepsiesHaley B Dame, Hema Kopalle, Belen Miñana, et al.
Neuron|October 22, 2013
RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense interventionChristopher J Donnelly, Ping-Wu Zhang, Jacqueline T Pham, et al.
Science Translational Medicine|May 14, 2025
Intra-amniotic antisense oligonucleotide treatment improves phenotypes in preclinical models of spinal muscular atrophyBeltran Borges, Stephen M Brown, Wan-Jin Chen, et al.
Nature Neuroscience|October 2, 2012
Divergent roles of ALS-linked proteins FUS/TLS and TDP-43 intersect in processing long pre-mRNAsClotilde Lagier-Tourenne, Magdalini Polymenidou, Kasey R Hutt, et al.
The New England Journal of Medicine|February 15, 2018
Nusinersen versus Sham Control in Later-Onset Spinal Muscular AtrophyEugenio Mercuri, Basil T Darras, Claudia A Chiriboga, et al.
The Journal of Clinical Investigation|October 8, 2019
Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatmentDaniel M Ramos, Constantin d'Ydewalle, Vijayalakshmi Gabbeta, et al.
The Journal of Clinical Investigation|July 17, 2018
Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS modelsAlex McCampbell, Tracy Cole, Amy J Wegener, et al.
Nucleic Acids Research|May 12, 2019
Conjugation of hydrophobic moieties enhances potency of antisense oligonucleotides in the muscle of rodents and non-human primatesMichael E Østergaard, Michaela Jackson, Audrey Low, et al.
Pageof 17