Christian L Lorson Selected Research

Spinal muscular atrophy with respiratory distress 1

1/2022	The Ighmbp2D564N mouse model is the first SMARD1 model to demonstrate respiratory defects.
12/2021	Defining the optimal dose and therapeutic window in SMA with respiratory distress type I model mice, FVB/NJ-Ighmpb2 nmd-2J.
12/2019	Development of a novel severe mouse model of spinal muscular atrophy with respiratory distress type 1: FVB-nmd.
1/2019	Muscle fiber-type selective propensity to pathology in the nmd mouse model of SMARD1.
9/2018	A Direct Comparison of IV and ICV Delivery Methods for Gene Replacement Therapy in a Mouse Model of SMARD1.
1/2018	Selective vulnerability in neuronal populations in nmd/SMARD1 mice.
1/2016	Rescue of a Mouse Model of Spinal Muscular Atrophy With Respiratory Distress Type 1 by AAV9-IGHMBP2 Is Dose Dependent.

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Christian L Lorson Research Topics

Disease

20	Spinal Muscular Atrophy (Progressive Muscular Atrophy) 01/2022 - 01/2002
8	Neurodegenerative Diseases (Neurodegenerative Disease) 01/2022 - 09/2011
7	Spinal muscular atrophy with respiratory distress 1 01/2022 - 01/2016
4	COVID-19 11/2021 - 04/2021
4	Inborn Genetic Diseases (Disease, Hereditary) 12/2012 - 01/2008
4	Ewing Sarcoma (Sarcoma, Ewing) 06/2010 - 11/2003
2	Infections 01/2022 - 10/2021
2	Motor Neuron Disease (Primary Lateral Sclerosis) 01/2019 - 01/2017
2	Atrophy 01/2019 - 04/2015
2	Huntington Disease (Huntington's Disease) 01/2017 - 12/2012
1	Inflammation (Inflammations) 11/2022
1	Severe Acute Respiratory Syndrome 04/2021
1	Hydrocephalus (Hydrocephaly) 12/2019
1	Body Weight (Weight, Body) 01/2017
1	Muscular Atrophy (Muscle Atrophy) 04/2015
1	Spinal Diseases (Disease, Spinal) 04/2015
1	Weight Gain 09/2014
1	Neuromuscular Diseases (Neuromuscular Disease) 12/2009
1	Autonomic Nervous System Diseases (Dysautonomia) 01/2008
1	Virus Diseases (Viral Diseases) 04/2002
1	Parvoviridae Infections (Parvovirus Infection) 04/2002

Drug/Important Bio-Agent (IBA)

19	Proteins (Proteins, Gene)FDA Link 01/2022 - 01/2002
3	RNA (Ribonucleic Acid)IBA 01/2017 - 12/2009
2	Antiviral Agents (Antivirals)IBA 11/2021 - 04/2021
2	remdesivirIBA 11/2021 - 04/2021
2	Pharmaceutical PreparationsIBA 11/2021 - 07/2013
2	Protein Isoforms (Isoforms)IBA 01/2018 - 04/2015
2	polyglutamineIBA 01/2017 - 12/2012
2	Antisense OligonucleotidesIBA 01/2017 - 01/2016
2	Small Nuclear Ribonucleoproteins (snRNP)IBA 04/2015 - 07/2011
2	RNA Precursors (Precursor, mRNA)IBA 12/2012 - 01/2010
2	RNA-Binding Protein EWSIBA 06/2010 - 12/2009
2	Glycine (Aminoacetic Acid)FDA LinkGeneric 06/2010 - 11/2003
2	Arginine (L-Arginine)FDA Link 06/2010 - 11/2003
1	Discoidin Domain ReceptorsIBA 01/2022
1	Viral ProteinsIBA 01/2022
1	Biological ProductsIBA 01/2022
1	Viral DNAIBA 01/2022
1	Carrier Proteins (Binding Protein)IBA 12/2021
1	Immunoglobulins (Immunoglobulin)IBA 12/2021
1	Glycoproteins (Glycoprotein)IBA 11/2021
1	VaccinesIBA 11/2021
1	Biomarkers (Surrogate Marker)IBA 10/2021
1	ElementsIBA 10/2021
1	alpha-SynucleinIBA 01/2017
1	Azithromycin (Zithromax)FDA LinkGeneric 01/2017
1	MorpholinosIBA 01/2016
1	Complementary DNA (cDNA)IBA 01/2016
1	MyostatinIBA 03/2012
1	Single-Stranded DNA (DNA, Single Stranded)IBA 12/2011
1	Nerve Growth Factors (Neurotrophins)IBA 02/2011
1	Staphylococcal Protein A (Protein A)IBA 06/2010
1	TC007IBA 10/2009
1	FollistatinIBA 03/2009
1	gallocatechol (epigallocatechin)IBA 01/2008
1	PolyphenolsIBA 01/2008
1	Messenger RNA (mRNA)IBA 01/2008
1	AminoglycosidesIBA 11/2006
1	Peptides (Polypeptides)IBA 01/2004
1	RNA-Binding Proteins (RNA-Binding Protein)IBA 11/2003
1	Transcription Factors (Transcription Factor)IBA 11/2003
1	Viral Nonstructural ProteinsIBA 04/2002

Therapy/Procedure

10	Therapeutics 12/2021 - 10/2008
2	Denervation 01/2019 - 01/2018
1	Highly Active Antiretroviral Therapy (HAART) 11/2022