List of works - Juan Jose Marugan

155. High Throughput screen for identifying small molecule modulators of alpha-glucosidase for the potential treatment of Pompe disease

5-(4-(4-Acetylphenyl)piperazin-1-ylsulfonyl)indolin-2-one Analogs as Inhibitors of Acid alpha-Glucosidase for Potential Chaperone Treatment of Pompe Disease or Intervention for Diabetes Mellitus Type 2

A high throughput glucocerebrosidase assay using the natural substrate glucosylceramide

scientific article

A high throughput screening assay system for the identification of small molecule inhibitors of gsp

scientific article

A high-throughput sphingomyelinase assay using natural substrate.

scientific article

A homogenous luminescence assay reveals novel inhibitors for giardia lamblia carbamate kinase

scientific article published on 31 December 2012

A novel brain penetrant NPS receptor antagonist, NCGC00185684, blocks alcohol-induced ERK-phosphorylation in the central amygdala and decreases operant alcohol self-administration in rats

scientific journal article

Agonist-specific voltage-dependent gating of lysosomal two-pore Na channels

scientific article published on 11 December 2019

Collaborative development of 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick type C1 disease

scientific article

Discovery and Development of Small Molecules That Reduce PNC Prevalence

Discovery of Small Molecule Influenza Virus NS1 Antagonist

Discovery of a novel noniminosugar acid α glucosidase chaperone series

scientific article

Discovery of novel antigiardiasis drug candidates

scientific article

Discovery, Optimization, and Characterization of Novel Chlorcyclizine Derivatives for the Treatment of Hepatitis C Virus Infection.

scientific article published on 24 November 2015

Discovery, SAR and Biological Evaluation of Aryl-thiazol-piperidines as SMN Modulators

scientific article published in October 2011

Discovery, SAR, and Biological Evaluation of Non-inhibitory Chaperones of Glucocerebrosidase

Discovery, SAR, and Biological Evaluation of a Non-Inhibitory Chaperone for Acid Alpha Glucosidase

Discovery, optimization, and biological activity of the first potent and selective small-molecule agonist series of human relaxin receptor 1 (RXFP1)

Discovery, optimization, and characterization of a novel series of dopamine D2 versus D3 receptor selective antagonists

Discovery, optimization, and characterization of novel D2 dopamine receptor selective antagonists

scientific article

Discovery, structure-activity relationship, and biological evaluation of noninhibitory small molecule chaperones of glucocerebrosidase

scientific article

Discovery, synthesis, and biological evaluation of novel SMN protein modulators

scientific article

Evaluation of 2-thioxo-2,3,5,6,7,8-hexahydropyrimido[4,5-d]pyrimidin-4(1H)-one analogues as GAA activators

scientific article

Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity

scientific article

HTS for Identification of Inhibitors against the ERK Signaling Pathway using a Homogenous Cell-based Assay

High throughput screening for SMA

High throughput screening for inhibitors of alpha-galactosidase

scientific article

High throughput screening for small molecule therapy for Gaucher disease using patient tissue as the source of mutant glucocerebrosidase

scientific article

High-Throughput Multiplexed Quantitation of Protein Aggregation and Cytotoxicity in a Huntington's Disease Model

scientific article

High-Throughput Screening, Discovery, and Optimization To Develop a Benzofuran Class of Hepatitis C Virus Inhibitors

scientific article published on 2 September 2015

High-throughput Giardia lamblia viability assay using bioluminescent ATP content measurements

scientific article

Identification and optimization of small-molecule agonists of the human relaxin hormone receptor RXFP1.

scientific article

Identification of 4-phenylquinolin-2(1H)-one as a specific allosteric inhibitor of Akt.

scientific article published on 15 September 2017

Identification of Functionally Selective Small Molecule Antagonists of the Neuropeptide-S Receptor: Naphthopyranopyrimidines

Identification of Modulators of the N370S Mutant Form of Glucocerebrosidase as a Potential Therapy for Gaucher Disease - Chemotype 1

Identification of Modulators of the N370S Mutant Form of Glucocerebrosidase as a Potential Therapy for Gaucher Disease - Chemotype 2

Identification of Potent and Selective Thyroid Stimulating Hormone Receptor Agonists

Identification of Small Molecule Antagonists of the Neuropeptide-S Receptor

Identification of Thyroid Stimulating Hormone Receptor Inverse Agonists

scientific article published on 10 May 2013

Identification of a selective small-molecule inhibitor series targeting the eyes absent 2 (Eya2) phosphatase activity

scientific article

Identification of benzodiazepine Ro5-3335 as an inhibitor of CBF leukemia through quantitative high throughput screen against RUNX1-CBFβ interaction

scientific article

Identification of compounds which inhibit cytotoxicity associated with mutant Huntingtin protein expression

Identification of novel anti-hepatitis C virus agents by a quantitative high throughput screen in a cell-based infection assay

scientific article published on 26 October 2015

Identification of small-molecule agonists of human relaxin family receptor 1 (RXFP1) by using a homogenous cell-based cAMP assay

scientific article

Induction and reversal of myotonic dystrophy type 1 pre-mRNA splicing defects by small molecules.

scientific article

Inhibition of PIP4Kγ ameliorates the pathological effects of mutant huntingtin protein.

scientific article published on 19 December 2017

Lomofungin and dilomofungin: inhibitors of MBNL1-CUG RNA binding with distinct cellular effects

scientific article

ML223: A Small Molecule Probe With In Vivo Activity Against Acute Myeloid Leukemia Subtype M4Eo

scientific article published on 10 May 2013

ML372 blocks SMN ubiquitination and improves spinal muscular atrophy pathology in mice

scientific article

Macrophage models of Gaucher disease for evaluating disease pathogenesis and candidate drugs

scientific article

Mannose receptor (CD206) activation in tumor-associated macrophages enhances adaptive and innate antitumor immune responses

scientific article published on 01 February 2020

Metarrestin, a perinucleolar compartment inhibitor, effectively suppresses metastasis.

scientific article published in May 2018

Methyl-β-cyclodextrin restores impaired autophagy flux in Niemann-Pick C1-deficient cells through activation of AMPK.

scientific article

Multi-gram scale synthesis of FR180204.

scientific article

Niemann-Pick Disease Type C: Induced Pluripotent Stem Cell-Derived Neuronal Cells for Modeling Neural Disease and Evaluating Drug Efficacy

scientific article

Non-iminosugar glucocerebrosidase small molecule chaperones

scientific article

Novel patient cell-based HTS assay for identification of small molecules for a lysosomal storage disease

scientific article

Repurposing of the antihistamine chlorcyclizine and related compounds for treatment of hepatitis C virus infection.

scientific article

SMN Modulator ML372 Increases SMN Protein Abundance, Body Weight, Lifespan, and Rescues Motor Function in SMNΔ7 SMA Mice

SU9516 Increases α7β1 Integrin and Ameliorates Disease Progression in the mdx Mouse Model of Duchenne Muscular Dystrophy

scientific article published on 5 April 2017

Selective Modulation of Gq/Gs pathways by Naphtho Pyrano Pyrimidines as antagonists of the Neuropeptide S Receptor

scientific article

Small Molecule Inhibitors of Activation-Induced Deaminase Decrease Class Switch Recombination in B Cells

scientific article published on 07 May 2021

Small-molecule activation of lysosomal TRP channels ameliorates Duchenne muscular dystrophy in mouse models

scientific article published on 07 February 2020

Structure-activity relationship of imidazopyridinium analogues as antagonists of neuropeptide s receptor

scientific article (publication date: 27 November 2013)

The pilot phase of the NIH Chemical Genomics Center

scientific article

Two high-throughput screening assays for aberrant RNA-protein interactions in myotonic dystrophy type 1.

scientific article

δ-Tocopherol reduces lipid accumulation in Niemann-Pick type C1 and Wolman cholesterol storage disorders

scientific article

δ-Tocopherol reduces lipid accumulation in Niemann-Pick type C1 and Wolman cholesterol storage disorders

scientific article published in Journal of Biological Chemistry

List of works by Juan Jose Marugan

155. High Throughput screen for identifying small molecule modulators of alpha-glucosidase for the potential treatment of Pompe disease

5-(4-(4-Acetylphenyl)piperazin-1-ylsulfonyl)indolin-2-one Analogs as Inhibitors of Acid alpha-Glucosidase for Potential Chaperone Treatment of Pompe Disease or Intervention for Diabetes Mellitus Type 2

A high throughput glucocerebrosidase assay using the natural substrate glucosylceramide

A high throughput screening assay system for the identification of small molecule inhibitors of gsp

A high-throughput sphingomyelinase assay using natural substrate.

A homogenous luminescence assay reveals novel inhibitors for giardia lamblia carbamate kinase

A novel brain penetrant NPS receptor antagonist, NCGC00185684, blocks alcohol-induced ERK-phosphorylation in the central amygdala and decreases operant alcohol self-administration in rats

Agonist-specific voltage-dependent gating of lysosomal two-pore Na channels

Collaborative development of 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick type C1 disease

Discovery and Development of Small Molecules That Reduce PNC Prevalence

Discovery of Small Molecule Influenza Virus NS1 Antagonist

Discovery of a novel noniminosugar acid α glucosidase chaperone series

Discovery of novel antigiardiasis drug candidates

Discovery, Optimization, and Characterization of Novel Chlorcyclizine Derivatives for the Treatment of Hepatitis C Virus Infection.

Discovery, SAR and Biological Evaluation of Aryl-thiazol-piperidines as SMN Modulators

Discovery, SAR, and Biological Evaluation of Non-inhibitory Chaperones of Glucocerebrosidase

Discovery, SAR, and Biological Evaluation of a Non-Inhibitory Chaperone for Acid Alpha Glucosidase

Discovery, optimization, and biological activity of the first potent and selective small-molecule agonist series of human relaxin receptor 1 (RXFP1)

Discovery, optimization, and characterization of a novel series of dopamine D2 versus D3 receptor selective antagonists

Discovery, optimization, and characterization of novel D2 dopamine receptor selective antagonists

Discovery, structure-activity relationship, and biological evaluation of noninhibitory small molecule chaperones of glucocerebrosidase

Discovery, synthesis, and biological evaluation of novel SMN protein modulators

Evaluation of 2-thioxo-2,3,5,6,7,8-hexahydropyrimido[4,5-d]pyrimidin-4(1H)-one analogues as GAA activators

Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity

HTS for Identification of Inhibitors against the ERK Signaling Pathway using a Homogenous Cell-based Assay

High throughput screening for SMA

High throughput screening for inhibitors of alpha-galactosidase

High throughput screening for small molecule therapy for Gaucher disease using patient tissue as the source of mutant glucocerebrosidase

High-Throughput Multiplexed Quantitation of Protein Aggregation and Cytotoxicity in a Huntington's Disease Model

High-Throughput Screening, Discovery, and Optimization To Develop a Benzofuran Class of Hepatitis C Virus Inhibitors

High-throughput Giardia lamblia viability assay using bioluminescent ATP content measurements

Identification and optimization of small-molecule agonists of the human relaxin hormone receptor RXFP1.

Identification of 4-phenylquinolin-2(1H)-one as a specific allosteric inhibitor of Akt.

Identification of Functionally Selective Small Molecule Antagonists of the Neuropeptide-S Receptor: Naphthopyranopyrimidines

Identification of Modulators of the N370S Mutant Form of Glucocerebrosidase as a Potential Therapy for Gaucher Disease - Chemotype 1

Identification of Modulators of the N370S Mutant Form of Glucocerebrosidase as a Potential Therapy for Gaucher Disease - Chemotype 2

Identification of Potent and Selective Thyroid Stimulating Hormone Receptor Agonists

Identification of Small Molecule Antagonists of the Neuropeptide-S Receptor

Identification of Thyroid Stimulating Hormone Receptor Inverse Agonists

Identification of a selective small-molecule inhibitor series targeting the eyes absent 2 (Eya2) phosphatase activity

Identification of benzodiazepine Ro5-3335 as an inhibitor of CBF leukemia through quantitative high throughput screen against RUNX1-CBFβ interaction

Identification of compounds which inhibit cytotoxicity associated with mutant Huntingtin protein expression

Identification of novel anti-hepatitis C virus agents by a quantitative high throughput screen in a cell-based infection assay

Identification of small-molecule agonists of human relaxin family receptor 1 (RXFP1) by using a homogenous cell-based cAMP assay

Induction and reversal of myotonic dystrophy type 1 pre-mRNA splicing defects by small molecules.

Inhibition of PIP4Kγ ameliorates the pathological effects of mutant huntingtin protein.

Lomofungin and dilomofungin: inhibitors of MBNL1-CUG RNA binding with distinct cellular effects

ML223: A Small Molecule Probe With In Vivo Activity Against Acute Myeloid Leukemia Subtype M4Eo

ML372 blocks SMN ubiquitination and improves spinal muscular atrophy pathology in mice

Macrophage models of Gaucher disease for evaluating disease pathogenesis and candidate drugs

Mannose receptor (CD206) activation in tumor-associated macrophages enhances adaptive and innate antitumor immune responses

Metarrestin, a perinucleolar compartment inhibitor, effectively suppresses metastasis.

Methyl-β-cyclodextrin restores impaired autophagy flux in Niemann-Pick C1-deficient cells through activation of AMPK.

Multi-gram scale synthesis of FR180204.

Niemann-Pick Disease Type C: Induced Pluripotent Stem Cell-Derived Neuronal Cells for Modeling Neural Disease and Evaluating Drug Efficacy

Non-iminosugar glucocerebrosidase small molecule chaperones

Novel patient cell-based HTS assay for identification of small molecules for a lysosomal storage disease

Repurposing of the antihistamine chlorcyclizine and related compounds for treatment of hepatitis C virus infection.

SMN Modulator ML372 Increases SMN Protein Abundance, Body Weight, Lifespan, and Rescues Motor Function in SMNΔ7 SMA Mice

SU9516 Increases α7β1 Integrin and Ameliorates Disease Progression in the mdx Mouse Model of Duchenne Muscular Dystrophy

Selective Modulation of Gq/Gs pathways by Naphtho Pyrano Pyrimidines as antagonists of the Neuropeptide S Receptor

Small Molecule Inhibitors of Activation-Induced Deaminase Decrease Class Switch Recombination in B Cells

Small-molecule activation of lysosomal TRP channels ameliorates Duchenne muscular dystrophy in mouse models

Structure-activity relationship of imidazopyridinium analogues as antagonists of neuropeptide s receptor

The pilot phase of the NIH Chemical Genomics Center

Two high-throughput screening assays for aberrant RNA-protein interactions in myotonic dystrophy type 1.

δ-Tocopherol reduces lipid accumulation in Niemann-Pick type C1 and Wolman cholesterol storage disorders

δ-Tocopherol reduces lipid accumulation in Niemann-Pick type C1 and Wolman cholesterol storage disorders