Neural stem cells for disease modeling of Wolman disease and evaluation of therapeutics

Induced Pluripotent Stem Cells for Disease Modeling and Evaluation of Therapeutics for Niemann-Pick Disease Type A

(2016) Yan Long et al.   Stem Cells Translational Medicine

A cost-effective and efficient reprogramming platform for large-scale production of integration-free human induced pluripotent stem cells in chemically defined culture

(2015) Jeanette Beers et al.   Scientific Reports

A platform for rapid generation of single and multiplexed reporters in human iPSC lines

(2015) Ying Pei et al.   Scientific Reports

Clinical effect of sebelipase alfa on survival and growth in infants with lysosomal acid lipase deficiency (Wolman disease)

(2014) Vassili Valayannopoulos et al.   MOLECULAR GENETICS AND METABOLISM

δ-Tocopherol Reduces Lipid Accumulation in Niemann-Pick Type C1 and Wolman Cholesterol Storage Disorders

(2012) Miao Xu et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

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(2012) Takahiro Tsukimura et al.   MOLECULAR MEDICINE

Passaging and colony expansion of human pluripotent stem cells by enzyme-free dissociation in chemically defined culture conditions

(2012) Jeanette Beers et al.   Nature Protocols

Lysosomal Acid Lipase Deficiency Impairs Regulation ofABCA1Gene and Formation of High Density Lipoproteins in Cholesteryl Ester Storage Disease

(2011) Kristin L. Bowden et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Endocytosis of beta-cyclodextrins is responsible for cholesterol reduction in Niemann-Pick type C mutant cells

(2010) A. I. Rosenbaum et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Long-term metabolic, endocrine and neuropsychological outcome of hematopoietic cell transplantation for Wolman disease

(2008) J Tolar et al.   BONE MARROW TRANSPLANTATION