Chester B Whitley Selected Research

Mucopolysaccharidosis I (Hurler Syndrome)

3/2021	Biomarkers for prediction of skeletal disease progression in mucopolysaccharidosis type I.
1/2020	A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I.
1/2020	Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation.
10/2019	Genotype-phenotype relationships in mucopolysaccharidosis type I (MPS I): Insights from the International MPS I Registry.
9/2019	A longitudinal study of neurocognition and behavior in patients with Hurler-Scheie syndrome heterozygous for the L238Q mutation.
1/2019	ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome.
11/2018	Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison.
1/2018	RTB lectin-mediated delivery of lysosomal α-l-iduronidase mitigates disease manifestations systemically including the central nervous system.
3/2017	Pilot study of the safety and effect of adalimumab on pain, physical function, and musculoskeletal disease in mucopolysaccharidosis types I and II.
1/2017	Proteomic analysis of mucopolysaccharidosis I mouse brain with two-dimensional polyacrylamide gel electrophoresis.

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Chester B Whitley Research Topics

Disease

24	Mucopolysaccharidosis I (Hurler Syndrome) 03/2021 - 06/2004
13	Mucopolysaccharidoses 01/2020 - 01/2002
5	Inflammation (Inflammations) 01/2022 - 02/2015
5	Gangliosidoses (Gangliosidosis) 12/2021 - 02/2015
5	Mucopolysaccharidosis III (Sanfilippo Syndrome) 09/2021 - 05/2014
5	Mucopolysaccharidosis VI (Syndrome, Maroteaux-Lamy) 12/2010 - 05/2004
4	Disease Progression 12/2021 - 01/2018
4	Mucopolysaccharidosis VII (Sly Syndrome) 09/2020 - 01/2018
4	Sandhoff Disease (Sandhoff's Disease) 09/2019 - 02/2015
4	Mucopolysaccharidosis II (Hunter Syndrome) 01/2018 - 01/2003
4	Phenylketonurias (Phenylketonuria) 02/2012 - 08/2004
3	Lysosomal Storage Diseases (Lysosomal Storage Disease) 01/2020 - 08/2008
3	Rare Diseases (Rare Disease) 01/2020 - 02/2016
3	Tay-Sachs Disease 09/2019 - 02/2015
3	Neurodegenerative Diseases (Neurodegenerative Disease) 01/2019 - 02/2015
3	Pain (Aches) 03/2017 - 01/2014
2	Gaucher Disease (Gaucher's Disease) 12/2021 - 02/2016
2	Wolman Disease (Wolman's Disease) 01/2020 - 11/2014
2	Mental Disorders (Mental Disorder) 09/2019 - 02/2014
2	Hypersensitivity (Allergy) 11/2014 - 05/2004
1	Contracture 03/2021
1	Hip Dislocation 03/2021
1	Cholecystitis 01/2020
1	Cholelithiasis 01/2020
1	Metabolic Diseases (Metabolic Disease) 01/2019
1	Atrophy 01/2019
1	Mucopolysaccharidosis IV (Morquio Syndrome) 01/2019
1	Inborn Genetic Diseases (Disease, Hereditary) 01/2018
1	Musculoskeletal Diseases (Musculoskeletal Disease) 03/2017
1	Muscle Hypotonia (Hypotonia) 01/2017
1	Deglutition Disorders (Dysphagia) 01/2017
1	Seizures (Absence Seizure) 01/2017
1	Glycogen Storage Disease Type II (Pompe's Disease) 09/2016
1	Hypothyroidism 09/2016
1	Chronic Pain 04/2016
1	Ganglioneuroblastoma 01/2016
1	Coarse facial features 01/2016
1	Neuroblastoma 01/2016
1	Liver Failure 12/2015
1	Dyslipidemias (Dyslipidemia) 12/2015
1	GM1 Gangliosidosis (Gangliosidosis GM1) 02/2015
1	Autistic Disorder (Autism) 05/2014
1	Brain Diseases (Brain Disorder) 02/2014
1	Joint Diseases (Joint Disease) 02/2014
1	Splenomegaly 02/2014
1	Heart Diseases (Heart Disease) 02/2014
1	Hepatomegaly 02/2014

Drug/Important Bio-Agent (IBA)

17	EnzymesIBA 09/2019 - 12/2004
13	GlycosaminoglycansIBA 01/2022 - 01/2002
11	Iduronidase (alpha-L-Iduronidase)IBA 10/2019 - 06/2004
5	Biomarkers (Surrogate Marker)IBA 12/2021 - 01/2014
5	GangliosidesIBA 12/2020 - 02/2015
5	Pharmaceutical PreparationsIBA 01/2018 - 05/2004
5	N-Acetylgalactosamine-4-Sulfatase (Arylsulfatase B)IBA 12/2010 - 05/2004
4	vestronidase alfaIBA 09/2020 - 01/2018
3	Heparitin Sulfate (Heparan Sulfate)IBA 09/2021 - 01/2019
3	HydrolasesIBA 01/2019 - 01/2018
3	sapropterin (tetrahydrobiopterin)FDA Link 02/2012 - 08/2007
3	Phenylalanine (L-Phenylalanine)FDA Link 05/2009 - 08/2004
2	heparan sulfate sulfataseIBA 09/2021 - 01/2019
2	LipidsIBA 01/2020 - 11/2014
2	Transaminases (Aminotransferases)IBA 01/2020 - 11/2014
2	Sebelipase alfaIBA 01/2020 - 11/2014
2	GlucuronidaseIBA 01/2020 - 09/2019
2	Hexosaminidases (Hexosaminidase)IBA 01/2019 - 01/2019
2	Tumor Necrosis Factor-alpha (Tumor Necrosis Factor)IBA 03/2017 - 04/2016
2	AntibodiesIBA 01/2017 - 02/2016
1	ElastinIBA 01/2022
1	chitotriosidaseIBA 12/2021
1	CytokinesIBA 03/2021
1	CreatinineIBA 03/2021
1	Interleukin-6 (Interleukin 6)IBA 03/2021
1	Zinc Finger NucleasesIBA 01/2020
1	GalactosidasesIBA 01/2019
1	AlbuminsIBA 01/2019
1	alpha-N-acetyl-D-glucosaminidase (alpha-N-acetylglucosaminidase)IBA 01/2019
1	Iduronic Acid (Iduronate)IBA 01/2018
1	Adalimumab (Humira)FDA Link 03/2017
1	polyacrylamideIBA 01/2017
1	idursulfase FDA Link 01/2017
1	Dermatan SulfateIBA 01/2017
1	Proteins (Proteins, Gene)FDA Link 01/2017
1	UbiquitinIBA 01/2017
1	Thyroxine (Levothyroxine)FDA LinkGeneric 09/2016
1	GlycogenIBA 09/2016
1	GlucosidasesIBA 09/2016
1	AcidsIBA 09/2016
1	Cytochrome P-450 Enzyme System (Cytochrome P450)IBA 02/2016
1	Cholesterol Esters (Cholesteryl Esters)IBA 11/2014
1	Triglycerides (Triacylglycerol)IBA 11/2014
1	Phenobarbital (Luminal)FDA Link 05/2014
1	Human Growth Hormone (Saizen)FDA LinkGeneric 02/2014

Therapy/Procedure

17	Enzyme Replacement Therapy 09/2020 - 05/2004
14	Therapeutics 12/2021 - 01/2003
5	Cell Transplantation 01/2020 - 02/2013
4	Hematopoietic Stem Cell Transplantation 02/2014 - 02/2005
2	Transplantation 12/2015 - 05/2014
1	Enteral Nutrition (Feeding, Tube) 01/2017
1	Anesthesia 05/2016
1	Drug Therapy (Chemotherapy) 02/2016