Maguire, Casey A.

High levels of AAV vector integration into CRISPR-induced DNA breaks

Hanlon, K. S., Kleinstiver, B. P., Garcia, S. P., Zaborowski, M. P., Volak, A., Spirig, S. E., Muller, A., Sousa, A. A., Tsai, S. Q., Bengtsson, N. E., et al. (2019) · Nature Communications

Virus-based gene therapy researchCRISPR and Genetic Engineering

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Microvesicle-associated AAV Vector as a Novel Gene Delivery System

Maguire, C. A., Balaj, L., Sivaraman, S., Crommentuijn, M. H., Ericsson, M., Mincheva-Nilsson, L., Baranov, V., Gianni, D., Tannous, B. A., Sena-Esteves, M., et al. (2012) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 283

Rescue of hearing by gene delivery to inner-ear hair cells using exosome-associated AAV

György, B., Sage, C., Indzhykulian, A. A., Scheffer, D. I., Brisson, A. R., Tan, S., Wu, X., Volak, A., Mu, D., Tamvakologos, P. I., et al. (2017) · Molecular Therapy

Virus-based gene therapy researchHearing, Cochlea, Tinnitus, Genetics

Cited by 243

Viral vectors for therapy of neurologic diseases

Choudhury, S. R., Hudry, E., Maguire, C. A., Sena-Esteves, M., Breakefield, X. O., & Grandi, P. (2017) · Neuropharmacology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 190

Exosome-associated AAV2 vector mediates robust gene delivery into the murine retina upon intravitreal injection

Wassmer, S. J., Carvalho, L. S., György, B., Vandenberghe, L. H., & Maguire, C. A. (2017) · Scientific Reports

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 164

Naturally enveloped AAV vectors for shielding neutralizing antibodies and robust gene delivery in vivo

György, B., Fitzpatrick, Z., Crommentuijn, M. H. W., Mu, D., & Maguire, C. A. (2014) · Biomaterials

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 153

Selection of an Efficient AAV Vector for Robust CNS Transgene Expression

Hanlon, K. S., Meltzer, J. C., Buzhdygan, T., Cheng, M. J., Sena-Esteves, M., Bennett, R. E., Sullivan, T. P., Razmpour, R., Gong, Y., Ng, C., et al. (2019) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 148

Exosome-associated AAV vector as a robust and convenient neuroscience tool

Hudry, E., Martin, C., Gandhi, S., György, B., Scheffer, D. I., Mu, D., Merkel, S. F., Mingozzi, F., Fitzpatrick, Z., Dimant, H., et al. (2016) · Gene Therapy

Virus-based gene therapy researchExtracellular vesicles in disease

Cited by 146

Trafficking of adeno‐associated virus vectors across a model of the blood–brain barrier; a comparative study of transcytosis and transduction using primary human brain endothelial cells

Merkel, S. F., Andrews, A. M., Lutton, E. M., Mu, D., Hudry, E., Hyman, B. T., Maguire, C. A., & Ramirez, S. H. (2017) · Journal of Neurochemistry

Barrier Structure and Function StudiesVirus-based gene therapy research

Cited by 132

Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors

Meliani, A., Boisgerault, F., Fitzpatrick, Z., Marmier, S., Leborgne, C., Collaud, F., Simon Sola, M., Charles, S., Ronzitti, G., Vignaud, A., et al. (2017) · Blood Advances

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 127

Efficient Gene Transfer to the Central Nervous System by Single-Stranded Anc80L65

Hudry, E., Andres-Mateos, E., Lerner, E. P., Volak, A., Cohen, O., Hyman, B. T., Maguire, C. A., & Vandenberghe, L. H. (2018) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 84

Adenoassociated virus serotype 9-mediated gene therapy for x-linked adrenoleukodystrophy

Gong, Y., Mu, D., Prabhakar, S., Moser, A., Musolino, P., Ren, J., Breakefield, X. O., Maguire, C. A., & Eichler, F. S. (2015) · Molecular Therapy

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 74

Intrathecal Adeno-Associated Viral Vector-Mediated Gene Delivery for Adrenomyeloneuropathy

Gong, Y., Berenson, A., Laheji, F., Gao, G., Wang, D., Ng, C., Volak, A., Kok, R., Kreouzis, V., Dijkstra, I. M., et al. (2019) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 45

Gene therapy for tuberous sclerosis complex type 2 in a mouse model by delivery of AAV9 encoding a condensed form of tuberin

Cheah, P.-S., Prabhakar, S., Yellen, D., Beauchamp, R. L., Zhang, X., Kasamatsu, S., Bronson, R. T., Thiele, E. A., Kwiatkowski, D. J., Stemmer-Rachamimov, A., et al. (2021) · Science Advances

Tuberous Sclerosis Complex ResearchVirus-based gene therapy research

Cited by 38

Role of Basal Forebrain Neurons in Adrenomyeloneuropathy in Mice and Humans

Gong, Y., Laheji, F., Berenson, A., Li, Y., Moser, A., Qian, A., Frosch, M., Sadjadi, R., Hahn, R., Maguire, C. A., et al. (2024) · Annals of Neurology

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 11

An in vitro and in vivo efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs

Vasireddy, V., Maguire, C. A., Anderson, D. W., Ng, C., Gong, Y., Eichler, F., Fourcade, S., Guilera, C., Onieva, A., Sanchez, A., et al. (2024) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 6