Karen Kozarsky

Io Therapeutics (United States) — United States

Biochemistry, Genetics and Molecular Biology · Life Sciences

43h-index8.7kcitations99works6.52yr avg
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Research Topics

Virus-based gene therapy research(30), Lysosomal Storage Disorders Research(19), CRISPR and Genetic Engineering(14), Cholesterol and Lipid Metabolism(12), Glycosylation and Glycoproteins Research(11)

Publications99 total

An in vitro and in vivo efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs
Molecular Therapy — Methods & Clinical Development·2024· 4 citedOpen Access
In vivo selection in non-human primates identifies AAV capsids for on-target CSF delivery to spinal cord
Molecular Therapy·2024· 9 citedOpen Access
<i>In vivo</i> selection in non-human primates identifies superior AAV capsids for on-target CSF delivery to spinal cord
2023· 2 citedOpen Access
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
Molecular Genetics and Metabolism Reports·2023· 6 citedOpen Access
Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction
Human Gene Therapy·2022· 8 citedOpen Access
Optimization of intrathecal delivery of an infused AAV9 vector for delivery of a gene therapy candidate for Adrenomyeloneuropathy (AMN) in Non-Human Primates (NHP) (P12-13.008)
Neurology·2022
Preclinical Pharmacology and Toxicology of intrathecally infused AAV9-hABCD1, a gene therapy candidate for AMN, in Non-Human Primates (S29.002)
Neurology·2022
An AAV Encoding Human ABCD1 Shows Dose-Responsive Expression and Function Following Spinal Cord Delivery in a Rodent Model of Adrenomyeloneuropathy (P1-1.Virtual)
Neurology·2022
Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I
Frontiers in Molecular Neuroscience·2021· 41 citedOpen Access
Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10
Molecular Genetics and Metabolism Reports·2020· 18 citedOpen Access
Strategic &amp; regulatory insights for gene therapy preclinical-clinical translation in CNS
Cell and Gene Therapy Insights·2019
AAV8-antiVEGFfab Ocular Gene Transfer for Neovascular Age-Related Macular Degeneration
Molecular Therapy·2017· 55 citedOpen Access
RGX-314, an AAV8 expressing an anti-VEGF protein, strongly suppresses subretinal neovascularization and vascular leakage in mouse models
2017· 1 cited
Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System–Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer
Human Gene Therapy·2017· 48 cited
Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I
Human Gene Therapy·2017· 64 citedOpen Access
Relative effectiveness of different routes of AAV administration for gene therapy of mucopolysaccharidosis
Molecular Genetics and Metabolism·2017
Neurologic improvement in a mouse model of Hunter syndrome (mucopolysaccharidosis type II) by treatment with AAV9 vector after the development of cognitive dysfunction
Molecular Genetics and Metabolism·2017
Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice
Human Gene Therapy·2016· 42 cited
346. AAV9 Delivery into Cerebrospinal Fluid Corrects CNS Disease in a Murine Model of Mucopolysaccharidosis Type II
Molecular Therapy·2016Open Access
25. Intranasal Gene Delivery of AAV Iduronidase: An Effective and Non-Invasive Approach for Treatmentof CNS Disease in a Murine Model of MPS Type I
Molecular Therapy·2016Open Access
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Frequent Co-authors

Monty Krieger(16), James M. Wilson(11), Attilio Rigotti(5), David M. Kingsley(5), Mary H. Donahee(4), Steven E. Raper(4), Mariann Grossman(3), Dudley K. Strickland(3), Wolfgang Sattler(3), Ernst Malle(3), Kelly M. Podetz-Pedersen(3), Walter C. Low(3), R. Scott McIvor(3), Robert Sege(3), Edward D. Allen(2), Evan A. Stein(2), John F. Engelhardt(2), David W.M. Muller(2), Paul J. Lupien(2), Daniel J. Rader(2)