Karen Kozarsky

基本信息

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职业迁徙

个人简介

Creative gene therapy scientist with expertise in developing gene therapies in large pharma and small biotech. Specializing in preclinical development of novel adeno-associated virus (AAV) vectors for unmet needs in orphan/rare diseases; integrating with long-term planning, including pipeline development, coordinating preclinical and clinical development paths, and portfolio and intellectual property (IP) development.

研究兴趣

论文共 74 篇作者统计合作学者相似作者

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In Vivo Selection in Non-Human Primates Identifies Superior AAV Capsids for On-Target CSF Delivery to Spinal Cord

Killian S. Hanlon,Ming Cheng, Roberto Montoro Ferrer, Jae Ryun Ryu,Boram Lee,Demitri de la Cruz,Nikita Patel,Paula Espinoza,Miguel C. Santoscoy,Yi Gong,Carrie Ng, Diane M. Nguyen,Josette Nammour,Sean W. Clark,Vivi M. Heine,Woong Sun,Karen Kozarsky,Casey A. Maguire

MOLECULAR THERAPY（2024）

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In Vivo Selection in Non-Human Primates Identifies AAV Capsids Having Enhanced Transgene Expression and Biodistribution in Spinal Cord after Intrathecal Administration

Casey A. Maguire,Killian S. Hanlon,Karen Kozarsky,Sean Clark, Yi Gong, Ming Cheng,Demitri De la Cruz,Nikita Patel,Miguel Santoscoy, Diane M. Nguyen,Josette Nammour

MOLECULAR THERAPYno. 4 (2023): 235-235

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Non-invasive Intravenous Administration of AAV9 Transducing Iduronate Sulfatase Leads to Global Metabolic Correction and Prevention of Neurologic Deficits in a Mouse Model of Hunter Syndrome

Kanut Laoharawee,Kelly M. Podetz-Pedersen,Tam T. Nguyen,Sajya M. Singh,Miles C. Smith,Lalitha R. Belur, Walter C. Low,Karen F. Kozarsky,R. Scott McIvor

MOLECULAR GENETICS AND METABOLISM REPORTS (2023)

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Optimization of Intrathecal Delivery of an Infused AAV9 Vector for Delivery of a Gene Therapy Candidate for Adrenomyeloneuropathy (AMN) in Non-Human Primates (NHP)

Vidyullatha Vasireddy,Sean W. Clark, David W. Anderson,Karen Kozarsky

MOLECULAR THERAPY（2022）

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Preclinical Pharmacology and Toxicology of Intrathecally Infused AAV9-hABCD1, a Gene Therapy Candidate for AMN, in Non-Human Primates (S29.002)

Vidyullatha Vasireddy, Spencer K. Clark, Mark E. Cartwright,Karen Kozarsky, David J. Anderson

Neurologyno. 18_supplement (2022)

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Preclinical Pharmacology and Toxicology of Intrathecally Infused AAV9-hABCD1 (SBT101), a Gene Therapy Candidate for AMN, in Non-Human Primates

Vidyullatha Vasireddy,Sean W. Clark,Mark Cartwright,Karen Kozarsky, David W. Anderson

MOLECULAR THERAPY（2022）

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An AAV Encoding Human ABCD1 Shows Dose-Responsive Expression and Function Following Spinal Cord Delivery in a Rodent Model of Adrenomyeloneuropathy (P1-1.Virtual)

Hee Kyung Park, Tony del Rio, Vidyullatha Vasireddy, Thomas Lutz, Carrie Ng, Yi Gong, Inge M. E. Dijkstra, Stephan Kemp, Casey A. Maguire, Spencer K. Clark, David E. Anderson,Karen Kozarsky

Neurologyno. 18_supplement (2022)

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An AAV Encoding Human ABCD1 Shows Dose-Responsive Expression and Function Following Spinal Cord Delivery in a Rodent Model of Adrenomyeloneuropathy