World's Best Scientists 2026 revealed!

D-Index & Metrics

Biology and Biochemistry

D-Index
65
Citations
23541
World Ranking
8968
National Ranking
3984

Research.com Recognitions

  • 2013 - Fellow of the American Association for the Advancement of Science (AAAS)

Overview

Brian K. Kaspar is affiliated with Insmed Incorporated in the United States, specializing in research spanning biochemistry, genetics, molecular biology, and medicine. Their work includes a focus on molecular biology and genetics within subfields such as cell biology, surgery, and endocrinology, diabetes, and metabolism.

The primary research topics Brian K. Kaspar has contributed to include:

  • Neurogenetic and Muscular Disorders Research
  • RNA modifications and cancer
  • Genetics and Neurodevelopmental Disorders
  • Muscle Physiology and Disorders
  • Muscle metabolism and nutrition
  • Thyroid Disorders and Treatments
  • RNA regulation and disease

The scientist has published extensively in several venues, with frequent contributions in:

  • Neurobiology of Aging
  • Molecular Therapy - Methods & Clinical Development
  • Cells
  • Thyroid
  • Molecular Therapy

Notable recent publications include:

  • "Neurotoxic Astrocytes Directly Converted from Sporadic and Familial ALS Patient Fibroblasts Reveal Signature Diversities and miR-146a Theragnostic Potential in Specific Subtypes," 2022, Cells
  • "Follistatin-induced muscle hypertrophy in aged mice improves neuromuscular junction innervation and function," 2021, Neurobiology of Aging
  • "AAV9-MCT8 Delivery at Juvenile Stage Ameliorates Neurological and Behavioral Deficits in a Mouse Model of MCT8-Deficiency," 2022, Thyroid
  • "Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates," 2023, Molecular Therapy
  • "Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 disease," 2023, Frontiers in Genetics

Their research collaborations feature frequent co-authors such as:

  • Kathrin Meyer
  • Shibi Likhite
  • Arthur H.M. Burghes
  • Chitra C. Iyer
  • Clive N. Svendsen

Brian K. Kaspar was awarded the title of Fellow of the American Association for the Advancement of Science (AAAS) in 2013, recognizing contributions to scientific research.

Best Publications

  • Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

    Kevin D Foust;Emily Nurre;Emily Nurre;Chrystal L Montgomery;Anna Hernandez

  • Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model.

    Brian K. Kaspar;Jerònia Lladó;Nushin Sherkat;Jeffrey D. Rothstein

  • Sonic hedgehog regulates adult neural progenitor proliferation in vitro and in vivo

    Karen Lai;Brian K. Kaspar;Fred H. Gage;David V. Schaffer

  • Astrocytes from familial and sporadic ALS patients are toxic to motor neurons

    Amanda M Haidet-Phillips;Mark E Hester;Carlos J Miranda;Kathrin Meyer

  • Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

    Kevin D Foust;Xueyong Wang;Vicki L McGovern;Lyndsey Braun

  • Cell culture. Progenitor cells from human brain after death.

    Theo D. Palmer;Philip H. Schwartz;Philippe Taupin;Brian Kaspar

  • Directed evolution of adeno-associated virus yields enhanced gene delivery vectors.

    Narendra Maheshri;James T Koerber;Brian K Kaspar;David V Schaffer

  • IGF-I instructs multipotent adult neural progenitor cells to become oligodendrocytes

    Jenny Hsieh;James B. Aimone;Brian K. Kaspar;Tomoko Kuwabara

  • Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders

    Adam K Bevan;Adam K Bevan;Sandra Duque;Kevin D Foust;Pablo R Morales

  • The C9orf72 protein interacts with Rab1a and the ULK1 complex to regulate initiation of autophagy.

    Christopher P Webster;Emma F Smith;Claudia S Bauer;Annekathrin Moller

  • Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS.

    Kathrin Meyer;Laura Ferraiuolo;Carlos J. Miranda;Shibi Likhite;Shibi Likhite

  • Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates

    Lluis Samaranch;Ernesto A. Salegio;Waldy San Sebastian;Adrian P. Kells

  • Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates

    Kathrin Meyer;Laura Ferraiuolo;Leah Schmelzer;Lyndsey Braun

  • Adeno-associated virus effectively mediates conditional gene modification in the brain

    Brian K. Kaspar;Bryce Vissel;Tasha Bengoechea;Steven Crone

  • A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy

    Jerry R Mendell;Jerry R Mendell;Zarife Sahenk;Zarife Sahenk;Vinod Malik;Ana M Gomez

  • Systemic delivery of MeCP2 rescues behavioral and cellular deficits in female mouse models of Rett syndrome.

    Saurabh K. Garg;Daniel T. Lioy;Daniel T. Lioy;Hélène Cheval;James C. McGann;James C. McGann

  • Early heart failure in the SMNΔ7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery

    Adam K. Bevan;Kirk R. Hutchinson;Kirk R. Hutchinson;Kevin D. Foust;Lyndsey Braun

  • A Conditional Deletion of the NR1 Subunit of the NMDA Receptor in Adult Spinal Cord Dorsal Horn Reduces NMDA Currents and Injury-Induced Pain

    Samantha M. South;Tatsuro Kohno;Brian K. Kaspar;Deborah Hegarty

  • Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS

    Kevin D Foust;Desirée L Salazar;Desirée L Salazar;Desirée L Salazar;Shibi Likhite;Shibi Likhite;Laura Ferraiuolo

  • Rapid and efficient generation of functional motor neurons from human pluripotent stem cells using gene delivered transcription factor codes.

    Mark E. Hester;Matthew J. Murtha;Sungwon Song;Meghan Rao

Frequent Co-Authors

Jerry R. Mendell
Jerry R. Mendell The Ohio State University
Arthur H.M. Burghes
Arthur H.M. Burghes The Ohio State University
Zarife Sahenk
Zarife Sahenk Nationwide Children's Hospital
John T. Kissel
John T. Kissel The Ohio State University
Don W. Cleveland
Don W. Cleveland University of California, San Diego
Thomas W. Prior
Thomas W. Prior The Ohio State University
Kevin M. Flanigan
Kevin M. Flanigan The Ohio State University
Richard S. Finkel
Richard S. Finkel St. Jude Children's Research Hospital
David V. Schaffer
David V. Schaffer University of California, Berkeley
Fred H. Gage
Fred H. Gage Salk Institute for Biological Studies

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