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Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 26, 2025
Age-sensitive response of systemic AAV-mediated gene therapy in a newly characterized feline model of mucolipidosis II
Nettie K Pyne, Jessica Bagel, Charles Shyng, et al.
Human Molecular Genetics
|
September 6, 2022
Functional assessment of homozygous ALDH18A1 variants reveals alterations in amino acid and antioxidant metabolism
Maxwell B Colonna, Tonya Moss, Sneha Mokashi, et al.
American Journal of Human Genetics
|
September 10, 2020
De Novo Variants in LMNB1 Cause Pronounced Syndromic Microcephaly and Disruption of Nuclear Envelope Integrity
Francesca Cristofoli, Tonya Moss, Hannah W Moore, et al.
Cell Reports. Medicine
|
May 31, 2023
Tracer metabolomics reveals the role of aldose reductase in glycosylation
Silvia Radenkovic, Anna N Ligezka, Sneha S Mokashi, et al.
Disease Models & Mechanisms
|
March 11, 2020
Deficiency in the endocytic adaptor proteins PHETA1/2 impairs renal and craniofacial development
Kristin M Ates, Tong Wang, Trevor Moreland, et al.
American Journal of Human Genetics
|
May 8, 2021
A mutation in SLC37A4 causes a dominantly inherited congenital disorder of glycosylation characterized by liver dysfunction
Bobby G Ng, Paulina Sosicka, François Fenaille, et al.
Kidney International
|
August 20, 2023
Certain heterozygous variants in the kinase domain of the serine/threonine kinase NEK8 can cause an autosomal dominant form of polycystic kidney disease
Laura R Claus, Chuan Chen, Jennifer Stallworth, et al.
Page
of 5
Search research articles
Search
Showing results (41-50 of 47) with videos related to
Sort By:
Page
of 5
You have reached the last page of results.
This site can display upto 47 results.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
April 26, 2025
Age-sensitive response of systemic AAV-mediated gene therapy in a newly characterized feline model of mucolipidosis II
Nettie K Pyne, Jessica Bagel, Charles Shyng, et al.
Human Molecular Genetics
|
September 6, 2022
Functional assessment of homozygous ALDH18A1 variants reveals alterations in amino acid and antioxidant metabolism
Maxwell B Colonna, Tonya Moss, Sneha Mokashi, et al.
American Journal of Human Genetics
|
September 10, 2020
De Novo Variants in LMNB1 Cause Pronounced Syndromic Microcephaly and Disruption of Nuclear Envelope Integrity
Francesca Cristofoli, Tonya Moss, Hannah W Moore, et al.
Cell Reports. Medicine
|
May 31, 2023
Tracer metabolomics reveals the role of aldose reductase in glycosylation
Silvia Radenkovic, Anna N Ligezka, Sneha S Mokashi, et al.
Disease Models & Mechanisms
|
March 11, 2020
Deficiency in the endocytic adaptor proteins PHETA1/2 impairs renal and craniofacial development
Kristin M Ates, Tong Wang, Trevor Moreland, et al.
American Journal of Human Genetics
|
May 8, 2021
A mutation in SLC37A4 causes a dominantly inherited congenital disorder of glycosylation characterized by liver dysfunction
Bobby G Ng, Paulina Sosicka, François Fenaille, et al.
Kidney International
|
August 20, 2023
Certain heterozygous variants in the kinase domain of the serine/threonine kinase NEK8 can cause an autosomal dominant form of polycystic kidney disease
Laura R Claus, Chuan Chen, Jennifer Stallworth, et al.
Page
of 5