Publications by authors named "Brian K Kaspar"

92Publications

Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease.

Mol Ther 2019 10 10;27(10):1836-1847. Epub 2019 Jul 10.

Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, USA; Department of Pediatrics, Sanford School of Medicine, University of South Dakota, Sioux Falls, SD 57069, USA. Electronic address:

View Article and Find Full Text PDF
October 2019

MRI-Guided Focused Ultrasound for Targeted Delivery of rAAV to the Brain.

Methods Mol Biol 2019 ;1950:177-197

Brain Sciences, Biological Sciences, Sunnybrook Research Institute, Toronto, ON, Canada.

View Article and Find Full Text PDF
July 2019

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.

N Engl J Med 2017 11;377(18):1713-1722

From the Center for Gene Therapy at the Research Institute at Nationwide Children's Hospital (J.R.M., S.A.-Z., L.R.R.-K., L.L., L.A., K.B., K.C., S.L., C.M., K.M., B.K.K.) and the Departments of Pediatrics (J.R.M., S.A.-Z., R.S., L.L., L.A., K.B., K.C., J.T.K., B.K.K.), Neurology (J.R.M., W.D.A., L.R.R.-K., A.H.M.B., B.K.K.), Pathology (T.W.P.), and Molecular and Cellular Biochemistry (A.H.M.B.), Ohio State University - both in Columbus; and AveXis, Bannockburn, IL (S.N., J.L., D.M.S., C.W., J.A.C., M.D.H., A.K., S.C., L.B., K.D.F., B.K.K.).

View Article and Find Full Text PDF
November 2017

HSPB1 mutations causing hereditary neuropathy in humans disrupt non-cell autonomous protection of motor neurons.

Exp Neurol 2017 11 7;297:101-109. Epub 2017 Aug 7.

Department of Biological Chemistry & Pharmacology, The Ohio State University Wexner Medical Center, Columbus, OH, United States; Department of Neurology, The Ohio State University Wexner Medical Center, Columbus, OH, United States. Electronic address:

View Article and Find Full Text PDF
November 2017

NEUROD1 Instructs Neuronal Conversion in Non-Reactive Astrocytes.

Stem Cell Reports 2017 06 11;8(6):1506-1515. Epub 2017 May 11.

Departments of Molecular Biology, Neurology and Neurotherapeutics, and Hamon Center for Regenerative Science and Medicine, UT Southwestern Medical Center, Dallas, TX 75390, USA. Electronic address:

View Article and Find Full Text PDF
June 2017

Additive amelioration of ALS by co-targeting independent pathogenic mechanisms.

Ann Clin Transl Neurol 2017 02 11;4(2):76-86. Epub 2017 Jan 11.

Center for Gene Therapy The Research Institute at Nationwide Children's Hospital Columbus Ohio; Biomedical Sciences Graduate Program College of Medicine The Ohio State University Columbus Ohio.

View Article and Find Full Text PDF
February 2017

High content analysis in amyotrophic lateral sclerosis.

Mol Cell Neurosci 2017 04 11;80:180-191. Epub 2016 Dec 11.

Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA; Department of Neuroscience, The Ohio State University, Columbus, OH, USA; Department of Pediatrics, College of Medicine and Public Health, The Ohio State University, Columbus, OH, USA. Electronic address:

View Article and Find Full Text PDF
April 2017

Oligodendrocytes contribute to motor neuron death in ALS via SOD1-dependent mechanism.

Proc Natl Acad Sci U S A 2016 10 29;113(42):E6496-E6505. Epub 2016 Sep 29.

The Research Institute, Nationwide Children's Hospital, Columbus, OH 43205; Department of Neuroscience, The Ohio State University, Columbus, OH 43210; Molecular, Cellular, and Developmental Biology Graduate Program, The Ohio State University, Columbus, OH 43210; Integrated Biomedical Science Graduate Program, College of Medicine, The Ohio State University, Columbus, OH 43210

View Article and Find Full Text PDF
October 2016

Potent spinal parenchymal AAV9-mediated gene delivery by subpial injection in adult rats and pigs.

Mol Ther Methods Clin Dev 2016 13;3:16046. Epub 2016 Jul 13.

Neuroregeneration Laboratory, Department of Anesthesiology, University of California, San Diego, California, USA; Institute of Neurobiology, Slovak Academy of Sciences, Kosice, Slovak Republic.

View Article and Find Full Text PDF
July 2016

Glia-neuron interactions in neurological diseases: Testing non-cell autonomy in a dish.

Brain Res 2017 02 9;1656:27-39. Epub 2016 Jan 9.

The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA; Molecular, Cellular & Developmental Biology Graduate Program, The Ohio State University, Columbus, OH, USA; Department of Neuroscience, The Ohio State University, Columbus, OH, USA. Electronic address:

View Article and Find Full Text PDF
February 2017