Louise R Rodino-Klapac Selected Research

Sarcoglycanopathies

1/2021	Preclinical Systemic Delivery of Adeno-Associated α-Sarcoglycan Gene Transfer for Limb-Girdle Muscular Dystrophy.
1/2019	Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion.
11/2010	Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D.
9/2009	Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins.

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Louise R Rodino-Klapac Research Topics

Disease

18	Duchenne Muscular Dystrophy (Muscular Dystrophy, Becker) 01/2021 - 09/2007
6	Fibrosis (Cirrhosis) 01/2021 - 06/2012
5	Muscular Dystrophies (Muscular Dystrophy) 01/2016 - 09/2009
4	Sarcoglycanopathies 01/2021 - 09/2009
3	Cardiomyopathies (Cardiomyopathy) 01/2017 - 03/2013
3	Muscular Diseases (Myopathy) 01/2016 - 11/2009
2	Limb-Girdle Muscular Dystrophies (Limb-Girdle Muscular Dystrophy) 01/2021 - 09/2009
2	Spinal Muscular Atrophy (Progressive Muscular Atrophy) 01/2021 - 11/2017
2	Dysferlinopathy 01/2018 - 03/2015
2	Muscle Weakness 05/2017 - 03/2015
2	Inflammation (Inflammations) 03/2015 - 06/2012
1	Retinitis Pigmentosa (Pigmentary Retinopathy) 01/2021
1	Severe combined immunodeficiency due to adenosine deaminase deficiency 01/2021
1	Alzheimer Disease (Alzheimer's Disease) 01/2021
1	Color Vision Defects (Color Blindness) 01/2021
1	Hemophilia A (Haemophilia) 01/2021
1	Hemorrhage 01/2021
1	Macular Degeneration (Age-Related Maculopathy) 01/2021
1	Necrosis 01/2021
1	Parkinson Disease (Parkinson's Disease) 01/2021
1	Choroideremia 01/2021
1	Severe Combined Immunodeficiency (Bare Lymphocyte Syndrome) 01/2021
1	Canavan Disease (Leukodystrophy, Spongiform) 01/2021
1	Congenital Structural Myopathies (Centronuclear Myopathy) 01/2021
1	Retinoschisis (Retinoschisis, Juvenile) 01/2021
1	Giant Axonal Neuropathy 01/2021
1	Leber Congenital Amaurosis 01/2021
1	Rare Diseases (Rare Disease) 01/2020
1	Disease Progression 06/2019
1	Neoplasms (Cancer) 01/2019
1	Facioscapulohumeral Muscular Dystrophy 03/2018
1	Motor Neuron Disease (Primary Lateral Sclerosis) 11/2017
1	Cicatrix (Scar) 05/2017
1	type 2E Limb-girdle muscular dystrophy 01/2017
1	Inclusion Body Myositis 01/2017
1	Respiratory Insufficiency (Respiratory Failure) 01/2017
1	Kyphosis 10/2015
1	Limb-girdle muscular dystrophy type 2A 05/2013
1	Scoliosis 06/2012

Drug/Important Bio-Agent (IBA)

13	DystrophinIBA 01/2021 - 09/2007
5	Sarcoglycans (beta Sarcoglycan)IBA 01/2021 - 09/2009
4	Proteins (Proteins, Gene)FDA Link 01/2021 - 09/2009
3	eteplirsenIBA 06/2019 - 11/2013
3	DysferlinIBA 01/2018 - 03/2015
3	FollistatinIBA 01/2017 - 01/2015
2	Codon (Codons)IBA 01/2020 - 11/2010
2	Protein Isoforms (Isoforms)IBA 01/2017 - 09/2015
2	MicroRNAs (MicroRNA)IBA 08/2013 - 05/2013
2	GlucocorticoidsIBA 03/2013 - 06/2012
1	tisagenlecleucelIBA 01/2021
1	axicabtagene ciloleucelIBA 01/2021
1	ZolgensmaIBA 01/2021
1	4- (4- dihexadecylaminostyryl)- N- methylpyridium (DiA)IBA 01/2021
1	Chimeric Antigen ReceptorsIBA 01/2021
1	Adenosine DeaminaseIBA 01/2021
1	Aromatic-L-Amino-Acid DecarboxylasesIBA 01/2021
1	Creatine Kinase (Creatine Phosphokinase)IBA 01/2021
1	Glycoproteins (Glycoprotein)IBA 01/2021
1	Fibronectins (Fibronectin)IBA 05/2017
1	integrin alpha7beta1IBA 10/2015
1	Carrier Proteins (Binding Protein)IBA 04/2014
1	Dystroglycans (Dystroglycan)IBA 04/2014
1	Laminin (Merosin)IBA 04/2014
1	MorpholinosIBA 11/2013
1	MyostatinIBA 06/2012
1	Nitric Oxide (Nitrogen Monoxide)FDA Link 06/2012
1	Phosphodiesterase InhibitorsIBA 06/2012
1	Complementary DNA (cDNA)IBA 01/2012
1	AminoglycosidesIBA 11/2010
1	Gentamicins (Gentamicin)FDA LinkGeneric 06/2010
1	Terminator Codon (Termination Codon)IBA 06/2010

Therapy/Procedure

5	Therapeutics 01/2021 - 11/2009
1	Drug Therapy (Chemotherapy) 01/2019
1	Artificial Respiration (Mechanical Ventilation) 11/2017
1	Intramuscular Injections 01/2017