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Publications by Mark L. Batshaw

174. Liver Fibrosis in Aged OTC-KO Heterozygotes and Successful Correction by AAV8-Mediated Gene Therapy

Molecular Therapy
Molecular MedicineMolecular BiologyPharmacologyMedicineGeneticsDrug Discovery
2015English

AAV Gene Therapy Corrects OTC Deficiency and Prevents Liver Fibrosis in Aged OTC-knock Out Heterozygous Mice

Molecular Genetics and Metabolism
GeneticsMolecular BiologyBiochemistryEndocrinologyMetabolismDiabetes
2017English

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Ocular and Systemic Safety of a Recombinant AAV8 Vector for X-Linked Retinoschisis Gene Therapy: GLP Studies in Rabbits and Rs1-Ko Mice

Molecular Therapy - Methods and Clinical Development
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Assessment of Toxicity and Biodistribution of Recombinant AAV8 Vector–mediated Immunomodulatory Gene Therapy in Mice With Pompe Disease

Molecular Therapy - Methods and Clinical Development
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2019English

Efficient Mitochondrial Import of Newly Synthesized Ornithine Transcarbamylase (OTC) and Correction of Secondary Metabolic Alterations in Spfash Mice Following Gene Therapy of OTC Deficiency

Molecular Medicine
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1999English

The Role of Apoptosis in Immune Hyporesponsiveness Following AAV8 Liver Gene Transfer

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2013English

Significant Alteration of Liver Metabolites by AAV8.Urocortin 2 Gene Transfer in Mice With Insulin Resistance

PLoS ONE
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2019English

Gene Therapy for Cystic Fibrosis

Chest
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1995English

Recombinant AAV8-mediated Intrastriatal Gene Delivery of CDNF Protects Rats Against Methamphetamine Neurotoxicity

International Journal of Medical Sciences
Medicine
2017English

Bronchoalveolar Fluid Is Not a Major Hindrance to Virus-Mediated Gene Therapy in Cystic Fibrosis

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