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ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome
Li Ou
, Russell C. DeKelver
, Michelle Rohde
, Susan Tom
, Robert Radeke
, Susan J. St. Martin
, Yolanda Santiago
, Scott Sproul
, Michelle J. Przybilla
,
Brenda L. Koniar
, Kelly M. Podetz-Pedersen
, Kanut Laoharawee
, Renee D. Cooksley
, Kathleen E. Meyer
, Michael C. Holmes
,
R. Scott McIvor
, Thomas Wechsler
,
Chester B. Whitley
Pediatric Genetics & Metabolism
Center for Translational Medicine
Genetics, Cell Biology and Development (TMED)
Administration (CBS)
Office of Academic Clinical Affairs
Genetics Mechanisms of Cancer
Research output
:
Contribution to journal
›
Article
›
peer-review
74
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Scopus citations
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Keyphrases
Hurler Syndrome
100%
Glycosaminoglycans
100%
α-L-iduronidase
100%
Photosystem II
100%
In Vivo Genome Editing
100%
Mucopolysaccharidosis 1
100%
Liver
50%
Enzyme Replacement Therapy
50%
Treatment Options
50%
Hepatocytes
50%
Murine Model
50%
Hematopoietic Stem Cell Transplantation
50%
Lysosomal Acid Lipase
50%
Severe Disease
50%
Mortality Risk
50%
Metabolic Disease
50%
Systemic Disease
50%
Albumin
50%
Current Treatment
50%
Corrective
50%
Morbidity Risk
50%
Monogenic Disease
50%
Enzyme Expression
50%
Substrate Accumulation
50%
Neurobehavioral Deficits
50%
Adequate Therapy
50%
Platform-based
50%
Shortened Lifespan
50%
Medicine and Dentistry
Hurler Syndrome
100%
Genome Editing
100%
Levo Iduronidase
50%
Enzyme Replacement Therapy
50%
Glycosaminoglycan
50%
Diseases
50%
Lifespan
25%
Protein Expression
25%
Water-Electrolyte Imbalance
25%
Secretion (Process)
25%
Hepatocyte
25%
Infusion
25%
Systemic Disease
25%
Hematopoietic Stem Cell Transplantation
25%
Albumin
25%
Hydrolase
25%
Treatment Option
25%
Biochemistry, Genetics and Molecular Biology
Genome Editing
100%
Hurler Syndrome
100%
Iduronidase
50%
Enzyme
50%
Glycosaminoglycan
50%
Hepatocyte
25%
Hydrolase
25%
Metabolic Disorder
25%
Lifespan
25%
Secretion (Process)
25%
Protein Expression
25%
Hematopoietic Stem Cell Transplantation
25%
Albumin
25%
Deficiency
25%
Pharmacology, Toxicology and Pharmaceutical Science
Hurler Syndrome
100%
Enzymes
75%
Levo Iduronidase
50%
Glycosaminoglycan
50%
Diseases
50%
Replacement Therapy
25%
Hydrolase
25%
Systemic Disease
25%
Albumin
25%
Metabolic Disorder
25%