Mark S Sands Selected Research

Globoid Cell Leukodystrophy (Krabbe Disease)

1/2022	CRISPR-Cas9 Knock-In of T513M and G41S Mutations in the Murine β-Galactosyl-Ceramidase Gene Re-capitulates Early-Onset and Adult-Onset Forms of Krabbe Disease.
1/2021	Substrate reduction therapy for Krabbe disease and metachromatic leukodystrophy using a novel ceramide galactosyltransferase inhibitor.
1/2021	Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease.
1/2021	Krabbe disease: New hope for an old disease.
1/2020	Krabbe disease successfully treated via monotherapy of intrathecal gene therapy.
1/2020	Cell-autonomous expression of the acid hydrolase galactocerebrosidase.
10/2019	Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target.
11/2016	Treatment for Krabbe's disease: Finding the combination.
11/2016	Hematopoietic Stem cell transplantation and lentiviral vector-based gene therapy for Krabbe's disease: Present convictions and future prospects.
4/2015	Mechanism-based combination treatment dramatically increases therapeutic efficacy in murine globoid cell leukodystrophy.

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Mark S Sands Research Topics

Disease

27	Lysosomal Storage Diseases (Lysosomal Storage Disease) 01/2021 - 12/2002
22	Globoid Cell Leukodystrophy (Krabbe Disease) 01/2022 - 07/2004
14	Neuronal Ceroid-Lipofuscinoses (Neuronal Ceroid Lipofuscinosis) 01/2017 - 09/2005
13	Mucopolysaccharidosis VII (Sly Syndrome) 01/2016 - 07/2002
10	Neurodegenerative Diseases (Neurodegenerative Disease) 01/2020 - 09/2005
9	Demyelinating Diseases (Demyelinating Disease) 01/2022 - 01/2007
5	Gliosis 01/2022 - 01/2007
5	Neuroinflammatory Diseases 01/2022 - 09/2012
4	Mucopolysaccharidoses 01/2021 - 04/2003
4	Disease Progression 01/2017 - 04/2004
3	Seizures (Absence Seizure) 01/2022 - 05/2008
3	Mucopolysaccharidosis III (Sanfilippo Syndrome) 01/2021 - 08/2007
3	Inflammation (Inflammations) 01/2020 - 10/2004
3	Central Nervous System Diseases (CNS Diseases) 10/2011 - 09/2005
2	Farber Lipogranulomatosis 10/2019 - 06/2013
2	Alzheimer Disease (Alzheimer's Disease) 10/2016 - 09/2014
2	Atrophy 11/2015 - 09/2014
2	Retinal Diseases 07/2008 - 07/2004
2	Neoplasms (Cancer) 07/2007 - 05/2006
2	Hemorrhage 12/2005 - 05/2003
1	Metachromatic Leukodystrophy (Sulfatide Lipidosis) 01/2021
1	Mucopolysaccharidosis I (Hurler Syndrome) 03/2018
1	Dementia (Dementias) 10/2016
1	Nervous System Diseases (Neurological Disorders) 10/2016
1	Rare Diseases (Rare Disease) 01/2016
1	Traumatic Brain Injuries (Traumatic Brain Injury) 09/2014
1	Tremor (Tremors) 01/2012
1	Leukoencephalopathies 12/2009
1	Body Weight (Weight, Body) 10/2009
1	Hypertrophy 10/2009
1	Atherosclerosis 06/2008
1	Blindness (Hysterical Blindness) 05/2008
1	Hepatocellular Carcinoma (Hepatoma) 07/2007
1	Thrombasthenia (Glanzmann Thrombasthenia) 12/2005
1	Osteoporosis 12/2005
1	Postmenopausal Osteoporosis 12/2005
1	Osteolysis 10/2004
1	Joint Diseases (Joint Disease) 05/2004

Drug/Important Bio-Agent (IBA)

25	EnzymesIBA 01/2022 - 09/2005
16	GalactosylceramidaseIBA 01/2021 - 09/2005
14	Proteins (Proteins, Gene)FDA Link 01/2021 - 09/2005
12	Psychosine (Galactosylsphingosine)IBA 01/2022 - 06/2010
11	GlucuronidaseIBA 01/2016 - 07/2002
7	Lysergic Acid Diethylamide (LSD)IBA 01/2021 - 06/2004
5	GlycosaminoglycansIBA 10/2016 - 04/2003
4	HydrolasesIBA 01/2021 - 07/2002
3	SphingolipidsIBA 01/2022 - 12/2013
3	alpha-N-acetyl-D-glucosaminidase (alpha-N-acetylglucosaminidase)IBA 01/2021 - 08/2007
3	Hexosaminidases (Hexosaminidase)IBA 01/2021 - 08/2007
3	AcidsIBA 01/2021 - 07/2002
3	palmitoyl-protein thioesteraseIBA 01/2017 - 01/2007
3	Retinaldehyde (Retinal)IBA 08/2015 - 07/2004
2	LipidsIBA 01/2022 - 11/2014
2	GlycolipidsIBA 01/2021 - 10/2019
2	Heparitin Sulfate (Heparan Sulfate)IBA 01/2021 - 10/2016
2	Galactosylceramides (Galactocerebrosides)IBA 01/2021 - 01/2021
2	VimentinIBA 10/2014 - 10/2011
2	CytokinesIBA 09/2014 - 10/2004
2	AntigensIBA 05/2006 - 05/2003
1	CeramidasesIBA 01/2022
1	CannabidiolIBA 01/2022
1	psychosine-3'-sulfate esterIBA 01/2021
1	N-Acylsphingosine GalactosyltransferaseIBA 01/2021
1	Sulfoglycosphingolipids (Sulfatides)IBA 01/2021
1	Acetylglucosaminidase (NAGase)IBA 01/2021
1	Acid CeramidaseIBA 10/2019
1	Iduronidase (alpha-L-Iduronidase)IBA 03/2018
1	apolipoprotein E1IBA 11/2016
1	Cholinergic Agents (Cholinergics)IBA 10/2016
1	CeroidIBA 11/2015
1	Kainic Acid (Kainate)IBA 09/2014
1	2- (4- (4- methyl- 6- phenylpyridazin- 3- yl)piperazin- 1- yl)pyrimidineIBA 09/2014
1	Membrane Proteins (Integral Membrane Proteins)IBA 11/2013
1	CeramidesIBA 06/2013
1	SphingosineIBA 06/2010
1	Indicators and Reagents (Reagents)IBA 12/2009
1	MonosaccharidesIBA 10/2009
1	Lipoproteins (Lipoprotein)IBA 06/2008
1	CholesterolIBA 06/2008
1	SterolsIBA 06/2008
1	Fatty Acids (Saturated Fatty Acids)IBA 05/2008
1	Aldehyde DehydrogenaseIBA 03/2008
1	LeptinIBA 12/2007
1	MicroRNAs (MicroRNA)IBA 07/2007
1	cadherin 5IBA 05/2006
1	Integrin beta3 (GPIIIa)IBA 12/2005
1	Integrin alphaVbeta3IBA 12/2005
1	IntegrinsIBA 12/2005
1	Tumor Necrosis Factor-alpha (Tumor Necrosis Factor)IBA 10/2004
1	Vascular Endothelial Growth Factor A (Vascular Endothelial Growth Factor)IBA 07/2004
1	Factor IX (Coagulation Factor IX)FDA LinkGeneric 05/2003

Therapy/Procedure

16	Therapeutics 01/2021 - 01/2003
8	Bone Marrow Transplantation (Transplantation, Bone Marrow) 01/2021 - 01/2004
5	Transplantation 11/2016 - 03/2008
4	Enzyme Replacement Therapy 03/2018 - 04/2004
3	Hematopoietic Stem Cell Transplantation 01/2021 - 11/2016
3	Heterologous Transplantation (Xenotransplantation) 07/2008 - 03/2003
1	Investigational Therapies (Experimental Therapy) 11/2014
1	Stem Cell Transplantation 09/2013
1	Blood Component Removal (Apheresis) 01/2004