Publications by authors named "Darin Falk"

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Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease.
David D Fuller, Sabhya Rana, Prajwal P Thakre, Ethan S Benevides, Megan K Pope, Darin J Falk

Mol Ther· September 2025

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Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease.
David D Fuller, Sabhya Rana, Prajwal Thakre, Ethan Benevides, Megan Pope, Darin J Falk

bioRxiv· December 2024

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Disease correction in mucopolysaccharidosis type IIIB mice by intraparenchymal or cisternal delivery of a capsid modified AAV8 codon-optimized NAGLU vector.
Courtney J Rouse, Kimberley Hawkins, Nadia Kabbej, Justin Dalugdug, Aishwarya Kunta, Darin J Falk

Hum Mol Genet· January 2023

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Reduction of Autophagic Accumulation in Pompe Disease Mouse Model Following Gene Therapy.
Angela L McCall, Sylvia G Stankov, Gabrielle Cowen, Denise Cloutier, Zizhao Zhang, Darin J Falk

Curr Gene Ther· July 2020

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AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the Hypoglossal Motor System of Pompe Mice.
Brendan M Doyle, Sara M F Turner, Michael D Sunshine, Phillip A Doerfler, Amy E Poirier, Darin J Falk

Mol Ther Methods Clin Dev· December 2019

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Pompe disease gene therapy: neural manifestations require consideration of CNS directed therapy.
Barry J Byrne, David D Fuller, Barbara K Smith, Nathalie Clement, Kirsten Coleman, Darin J Falk

Ann Transl Med· July 2019

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HSP90 Inhibitor, NVP-AUY922, Improves Myelination in Vitro and Supports the Maintenance of Myelinated Axons in Neuropathic Mice.
Vinita G Chittoor-Vinod, Hannah Bazick, Adrian G Todd, Darin Falk, Kathryn H Morelli

ACS Chem Neurosci· June 2019

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A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice.
Ni-Chung Lee, Wuh-Liang Hwu, Shin-Ichi Muramatsu, Darin J Falk, Barry J Byrne

Mol Neurobiol· June 2018

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Locomotor and skeletal muscle abnormalities in trembler J neuropathic mice.
Darin J Falk, Tori Galatas, Adrian G Todd, Elliott P Soto, Andrew B Harris

Muscle Nerve· April 2018

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Mechanical Ventilation for Duchenne Muscular Dystrophy: Sinner or Saint?
Barbara K Smith, Darin J Falk

Muscle Nerve· March 2018

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C/P MRS Metabolic Biomarkers of Disease Progression and Response to AAV Delivery of hGAA in a Mouse Model of Pompe Disease.
Celine Baligand, Adrian G Todd, Brittany Lee-McMullen, Ravneet S Vohra, Barry J Byrne, Darin J Falk

Mol Ther Methods Clin Dev· December 2017

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Neuropathology in respiratory-related motoneurons in young Pompe (Gaa(-/-)) mice.
Sara M F Turner, Aaron K Hoyt, Mai K ElMallah, Darin J Falk, Barry J Byrne

Respir Physiol Neurobiol· June 2016

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A scalable method for the production of high-titer and high-quality adeno-associated type 9 vectors using the HSV platform.
Laura Adamson-Small, Mark Potter, Darin J Falk, Brian Cleaver, Barry J Byrne

Mol Ther Methods Clin Dev· May 2016

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Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease.
Phillip A Doerfler, Adrian G Todd, Nathalie Clément, Darin J Falk, Sushrusha Nayak

Hum Gene Ther· January 2016

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Stimulation of Respiratory Motor Output and Ventilation in a Murine Model of Pompe Disease by Ampakines.
Mai K ElMallah, Silvia Pagliardini, Sara M Turner, Anthony J Cerreta, Darin J Falk

Am J Respir Cell Mol Biol· September 2015

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Endurance training ameliorates complex 3 deficiency in a mouse model of Barth syndrome.
Meghan S Soustek, Celine Baligand, Darin J Falk, Glenn A Walter, Alfred S Lewin

J Inherit Metab Dis· September 2015

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Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease.
Adrian G Todd, Jessica A McElroy, Robert W Grange, David D Fuller, Glenn A Walter, Darin J Falk

Ann Neurol· August 2015

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Altered activation of the tibialis anterior in individuals with Pompe disease: Implications for motor unit dysfunction.
Manuela Corti, Barbara K Smith, Darin J Falk, Lee Ann Lawson, David D Fuller

Muscle Nerve· June 2015

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Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe mice.
Darin J Falk, Meghan S Soustek, Adrian Gary Todd, Cathryn S Mah, Denise A Cloutier

Mol Ther Methods Clin Dev· June 2015

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Peripheral nerve and neuromuscular junction pathology in Pompe disease.
Darin J Falk, Adrian Gary Todd, Sooyeon Lee, Meghan S Soustek, Mai K ElMallah

Hum Mol Genet· February 2015

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Rapamycin improves peripheral nerve myelination while it fails to benefit neuromuscular performance in neuropathic mice.
Jessica Nicks, Sooyeon Lee, Andrew Harris, Darin J Falk, Adrian G Todd

Neurobiol Dis· October 2014

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Liposomes to target peripheral neurons and Schwann cells.
Sooyeon Lee, Ana Tari Ashizawa, Kwang Sik Kim, Darin J Falk, Lucia Notterpek

PLoS One· July 2014

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Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice.
Mai K Elmallah, Darin J Falk, Sushrusha Nayak, Roland A Federico, Milapjit S Sandhu

Mol Ther· April 2014

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Delivery of recombinant adeno-associated virus vectors to rat diaphragm muscle via direct intramuscular injection.
Ashley J Smuder, Darin J Falk, Kurt J Sollanek, W Bradley Nelson, Scott K Powers

Hum Gene Ther Methods· December 2013

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Adeno-associated virus-mediated gene therapy for metabolic myopathy.
Cathryn S Mah, Meghan S Soustek, A Gary Todd, Angela McCall, Barbara K Smith, Darin J Falk

Hum Gene Ther· November 2013

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