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Nature
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October 12, 2017
Radically truncated MeCP2 rescues Rett syndrome-like neurological defects
Rebekah Tillotson, Jim Selfridge, Martha V Koerner, et al.
Molecular Therapy. Methods & Clinical Development
|
May 13, 2017
Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome
Kamal K E Gadalla, Thishnapha Vudhironarit, Ralph D Hector, et al.
Molecular Therapy. Nucleic Acids
|
August 28, 2023
Long-term muscle-specific overexpression of DOK7 in mice using AAV9-tMCK-DOK7
Yu-Ting Huang, Hannah R Crick, Helena Chaytow, et al.
Molecular Therapy. Methods & Clinical Development
|
May 13, 2017
Improved <i>MECP2</i> Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery
Sarah E Sinnett, Ralph D Hector, Kamal K E Gadalla, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
November 24, 2011
High-resolution human cytomegalovirus transcriptome
Derek Gatherer, Sepehr Seirafian, Charles Cunningham, et al.
Neurology. Genetics
|
December 22, 2017
<i>CDKL5</i> variants: Improving our understanding of a rare neurologic disorder
Ralph D Hector, Vera M Kalscheuer, Friederike Hennig, et al.
Nature Communications
|
September 30, 2017
High-throughput RNA structure probing reveals critical folding events during early 60S ribosome assembly in yeast
Elena Burlacu, Fredrik Lackmann, Lisbeth-Carolina Aguilar, et al.
Science Translational Medicine
|
April 2, 2025
Self-regulating gene therapy ameliorates phenotypes and overcomes gene dosage sensitivity in a mouse model of Rett syndrome
Paul D Ross, Kamal K E Gadalla, Sophie R Thomson, et al.
Molecular Therapy. Nucleic Acids
|
July 2, 2026
Neonatal expression of human FMRP isoform corrects cortical deficits and improves behavior in a mouse model of fragile X syndrome
Anna O Norman, Courtney Scaramella, Dominik Biezonski, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 2, 2009
A novel model of SCID-X1 reconstitution reveals predisposition to retrovirus-induced lymphoma but no evidence of gammaC gene oncogenicity
Linda Scobie, Ralph D Hector, Louise Grant, et al.
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Search research articles
Search
Showing results (11-20 of 24) with videos related to
Sort By:
Page
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Nature
|
October 12, 2017
Radically truncated MeCP2 rescues Rett syndrome-like neurological defects
Rebekah Tillotson, Jim Selfridge, Martha V Koerner, et al.
Molecular Therapy. Methods & Clinical Development
|
May 13, 2017
Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome
Kamal K E Gadalla, Thishnapha Vudhironarit, Ralph D Hector, et al.
Molecular Therapy. Nucleic Acids
|
August 28, 2023
Long-term muscle-specific overexpression of DOK7 in mice using AAV9-tMCK-DOK7
Yu-Ting Huang, Hannah R Crick, Helena Chaytow, et al.
Molecular Therapy. Methods & Clinical Development
|
May 13, 2017
Improved <i>MECP2</i> Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery
Sarah E Sinnett, Ralph D Hector, Kamal K E Gadalla, et al.
Proceedings of the National Academy of Sciences of the United States of America
|
November 24, 2011
High-resolution human cytomegalovirus transcriptome
Derek Gatherer, Sepehr Seirafian, Charles Cunningham, et al.
Neurology. Genetics
|
December 22, 2017
<i>CDKL5</i> variants: Improving our understanding of a rare neurologic disorder
Ralph D Hector, Vera M Kalscheuer, Friederike Hennig, et al.
Nature Communications
|
September 30, 2017
High-throughput RNA structure probing reveals critical folding events during early 60S ribosome assembly in yeast
Elena Burlacu, Fredrik Lackmann, Lisbeth-Carolina Aguilar, et al.
Science Translational Medicine
|
April 2, 2025
Self-regulating gene therapy ameliorates phenotypes and overcomes gene dosage sensitivity in a mouse model of Rett syndrome
Paul D Ross, Kamal K E Gadalla, Sophie R Thomson, et al.
Molecular Therapy. Nucleic Acids
|
July 2, 2026
Neonatal expression of human FMRP isoform corrects cortical deficits and improves behavior in a mouse model of fragile X syndrome
Anna O Norman, Courtney Scaramella, Dominik Biezonski, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 2, 2009
A novel model of SCID-X1 reconstitution reveals predisposition to retrovirus-induced lymphoma but no evidence of gammaC gene oncogenicity
Linda Scobie, Ralph D Hector, Louise Grant, et al.
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of 3