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Frontiers in Medicine
|
May 15, 2026
rAAV9 vector biodistribution in nonhuman primate brain and spinal cord following lumbar intrathecal infusion
Emdadul Haque, Nino Devidze, Suku Nagendran, et al.
Molecular Therapy. Nucleic Acids
|
February 7, 2013
Optimization of scAAVIL-1ra In Vitro and In Vivo to Deliver High Levels of Therapeutic Protein for Treatment of Osteoarthritis
Laurie R Goodrich, Jennifer N Phillips, C Wayne McIlwraith, et al.
Experimental Eye Research
|
April 30, 2021
Intravitreal gene therapy protects against retinal dysfunction and degeneration in sheep with CLN5 Batten disease
Samantha J Murray, Katharina N Russell, Tracy R Melzer, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
May 27, 2010
Transient demyelination increases the efficiency of retrograde AAV transduction
Edmund R Hollis, Pouya Jamshidi, Ariana O Lorenzana, et al.
Molecular Therapy. Methods & Clinical Development
|
March 12, 2016
Construction of a hybrid β-hexosaminidase subunit capable of forming stable homodimers that hydrolyze GM2 ganglioside in vivo
Michael B Tropak, Sayuri Yonekawa, Subha Karumuthil-Melethil, et al.
Communications Medicine
|
January 27, 2025
Preclinical use of a clinically-relevant scAAV9/SUMF1 vector for the treatment of multiple sulfatase deficiency
Maximiliano Presa, Rachel M Bailey, Somdatta Ray, et al.
Molecular Therapy. Methods & Clinical Development
|
January 11, 2024
Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study
Alex E Ryckman, Natalie M Deschenes, Brianna M Quinville, et al.
Frontiers in Cellular Neuroscience
|
March 26, 2025
Riboflavin transporter deficiency: <i>AAV9-SLC52A2</i> gene therapy as a new therapeutic strategy
Cecilia Mei, Valentina Magliocca, Xin Chen, et al.
JCI Insight
|
June 8, 2023
CNS-dominant human FMRP isoform rescues seizures, fear, and sleep abnormalities in Fmr1-KO mice
Hayes Wong, Alexander Wm Hooper, Hye Ri Kang, et al.
Human Gene Therapy
|
March 30, 2011
Comparison of adeno-associated viral vector serotypes for spinal cord and motor neuron gene delivery
Brooke R Snyder, Steven J Gray, Eric T Quach, et al.
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of 11
Search research articles
Search
Showing results (51-60 of 106) with videos related to
Sort By:
Page
of 11
Frontiers in Medicine
|
May 15, 2026
rAAV9 vector biodistribution in nonhuman primate brain and spinal cord following lumbar intrathecal infusion
Emdadul Haque, Nino Devidze, Suku Nagendran, et al.
Molecular Therapy. Nucleic Acids
|
February 7, 2013
Optimization of scAAVIL-1ra In Vitro and In Vivo to Deliver High Levels of Therapeutic Protein for Treatment of Osteoarthritis
Laurie R Goodrich, Jennifer N Phillips, C Wayne McIlwraith, et al.
Experimental Eye Research
|
April 30, 2021
Intravitreal gene therapy protects against retinal dysfunction and degeneration in sheep with CLN5 Batten disease
Samantha J Murray, Katharina N Russell, Tracy R Melzer, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
May 27, 2010
Transient demyelination increases the efficiency of retrograde AAV transduction
Edmund R Hollis, Pouya Jamshidi, Ariana O Lorenzana, et al.
Molecular Therapy. Methods & Clinical Development
|
March 12, 2016
Construction of a hybrid β-hexosaminidase subunit capable of forming stable homodimers that hydrolyze GM2 ganglioside in vivo
Michael B Tropak, Sayuri Yonekawa, Subha Karumuthil-Melethil, et al.
Communications Medicine
|
January 27, 2025
Preclinical use of a clinically-relevant scAAV9/SUMF1 vector for the treatment of multiple sulfatase deficiency
Maximiliano Presa, Rachel M Bailey, Somdatta Ray, et al.
Molecular Therapy. Methods & Clinical Development
|
January 11, 2024
Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study
Alex E Ryckman, Natalie M Deschenes, Brianna M Quinville, et al.
Frontiers in Cellular Neuroscience
|
March 26, 2025
Riboflavin transporter deficiency: <i>AAV9-SLC52A2</i> gene therapy as a new therapeutic strategy
Cecilia Mei, Valentina Magliocca, Xin Chen, et al.
JCI Insight
|
June 8, 2023
CNS-dominant human FMRP isoform rescues seizures, fear, and sleep abnormalities in Fmr1-KO mice
Hayes Wong, Alexander Wm Hooper, Hye Ri Kang, et al.
Human Gene Therapy
|
March 30, 2011
Comparison of adeno-associated viral vector serotypes for spinal cord and motor neuron gene delivery
Brooke R Snyder, Steven J Gray, Eric T Quach, et al.
Page
of 11