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Clinical Genetics
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August 27, 2019
The genome empowerment scale: An assessment of parental empowerment in families with undiagnosed disease
Allyn McConkie-Rosell, Kelly Schoch, Jennifer Sullivan, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
August 6, 2002
Demonstration of feasibility of in vivo gene therapy for Gaucher disease using a chemically induced mouse model
John Marshall, Kerry Anne McEachern, Julie A Cavanagh Kyros, et al.
Blood
|
November 21, 2007
Characterization of in vitro antimurine thymocyte globulin-induced regulatory T cells that inhibit graft-versus-host disease in vivo
Melanie C Ruzek, James S Waire, Deborah Hopkins, et al.
The Journal of Urology
|
January 23, 2010
Stone forming risk factors in patients with type Ia glycogen storage disease
Charles D Scales, Aravind S Chandrashekar, Marnie R Robinson, et al.
Human Mutation
|
July 3, 2019
Heterozygous variants in MYBPC1 are associated with an expanded neuromuscular phenotype beyond arthrogryposis
Vandana Shashi, Janelle Geist, Youngha Lee, et al.
American Journal of Human Genetics
|
March 6, 2026
Newborn screening for type 1 diabetes using genome-based risk scores in the Early Check program
Nathan C Gaddis, Katerina S Kucera, Heidi L Cope, et al.
Open Forum Infectious Diseases
|
October 11, 2021
Demographics and Clinical Characteristics of Adult Patients Hospitalized due to COVID-19 in a Rural/Suburban Integrated Health System in Southcentral Pennsylvania, March Through May 2020
Michael A Bohrn, Ronald Benenson, Chelsea M Bush, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
July 31, 2018
Extensive Transduction and Enhanced Spread of a Modified AAV2 Capsid in the Non-human Primate CNS
Jerusha Naidoo, Lisa M Stanek, Kousaku Ohno, et al.
Human Molecular Genetics
|
November 20, 2024
De novo missense variants in the PP2A regulatory subunit PPP2R2B in a neurodevelopmental syndrome: potential links to mitochondrial dynamics and spinocerebellar ataxias
Priyanka Sandal, Chian Ju Jong, Ronald A Merrill, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
September 4, 2025
Development of an AAV-delivered microRNA gene therapy for Myotonic Dystrophy Type 1
Giulio S Tomassy, Wei Fan, Shuwen Cao, et al.
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of 6
Search research articles
Search
Showing results (41-50 of 60) with videos related to
Sort By:
Page
of 6
Clinical Genetics
|
August 27, 2019
The genome empowerment scale: An assessment of parental empowerment in families with undiagnosed disease
Allyn McConkie-Rosell, Kelly Schoch, Jennifer Sullivan, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
August 6, 2002
Demonstration of feasibility of in vivo gene therapy for Gaucher disease using a chemically induced mouse model
John Marshall, Kerry Anne McEachern, Julie A Cavanagh Kyros, et al.
Blood
|
November 21, 2007
Characterization of in vitro antimurine thymocyte globulin-induced regulatory T cells that inhibit graft-versus-host disease in vivo
Melanie C Ruzek, James S Waire, Deborah Hopkins, et al.
The Journal of Urology
|
January 23, 2010
Stone forming risk factors in patients with type Ia glycogen storage disease
Charles D Scales, Aravind S Chandrashekar, Marnie R Robinson, et al.
Human Mutation
|
July 3, 2019
Heterozygous variants in MYBPC1 are associated with an expanded neuromuscular phenotype beyond arthrogryposis
Vandana Shashi, Janelle Geist, Youngha Lee, et al.
American Journal of Human Genetics
|
March 6, 2026
Newborn screening for type 1 diabetes using genome-based risk scores in the Early Check program
Nathan C Gaddis, Katerina S Kucera, Heidi L Cope, et al.
Open Forum Infectious Diseases
|
October 11, 2021
Demographics and Clinical Characteristics of Adult Patients Hospitalized due to COVID-19 in a Rural/Suburban Integrated Health System in Southcentral Pennsylvania, March Through May 2020
Michael A Bohrn, Ronald Benenson, Chelsea M Bush, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
July 31, 2018
Extensive Transduction and Enhanced Spread of a Modified AAV2 Capsid in the Non-human Primate CNS
Jerusha Naidoo, Lisa M Stanek, Kousaku Ohno, et al.
Human Molecular Genetics
|
November 20, 2024
De novo missense variants in the PP2A regulatory subunit PPP2R2B in a neurodevelopmental syndrome: potential links to mitochondrial dynamics and spinocerebellar ataxias
Priyanka Sandal, Chian Ju Jong, Ronald A Merrill, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
September 4, 2025
Development of an AAV-delivered microRNA gene therapy for Myotonic Dystrophy Type 1
Giulio S Tomassy, Wei Fan, Shuwen Cao, et al.
Page
of 6