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A Single Intravenous Injection of AAV-PHP.B-hNDUFS4 Ameliorates the Phenotype of Ndufs4 Mice

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Adeno-associated virus–based gene therapy treats inflammatory kidney disease in mice

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Gene replacement therapy provides benefit in an adult mouse model of Leigh syndrome

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AAV-PHP.eB transduces both the inner and outer retina with high efficacy in mice

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Intramuscular Delivery of Gene Therapy for Targeting the Nervous System

Tosolini, A. P., & Sleigh, J. N. (2020) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2020.00129 · Cited by 32

Adeno-Associated Viral Vectors as Versatile Tools for Parkinson’s Research, Both for Disease Modeling Purposes and for Therapeutic Uses

Fajardo-Serrano, A., Rico, A. J., Roda, E., Honrubia, A., Arrieta, S., Ariznabarreta, G., Chocarro, J., Lorenzo-Ramos, E., Pejenaute, A., Vázquez, A., et al. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22126389 · Cited by 32

In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model

Chen, Y. A., Kankel, M. W., Hana, S., Lau, S. K., Zavodszky, M. I., McKissick, O. P., Mastrangelo, N., Dion, J., Wang, B., Ferretti, D., et al. (2022) · Gene Therapy · doi:10.1038/s41434-022-00375-w · Cited by 34

Drug Delivery Strategies to Overcome the Blood–Brain Barrier (BBB)

Helms, H. C. C., Kristensen, M., Saaby, L., Fricker, G., & Brodin, B. (2020) · Handbook of experimental pharmacology · doi:10.1007/164_2020_403 · Cited by 30

Programmable Assembly of Adeno-Associated Virus–Antibody Composites for Receptor-Mediated Gene Delivery

Zdechlik, A. C., He, Y., Aird, E. J., Gordon, W. R., & Schmidt, D. (2019) · Bioconjugate Chemistry · doi:10.1021/acs.bioconjchem.9b00790 · Cited by 30

Transgene distribution and immune response after ultrasound delivery of rAAV9 and PHP.B to the brain in a mouse model of amyloidosis

Kofoed, R. H., Heinen, S., Silburt, J., Dubey, S., Dibia, C. L., Maes, M. S., Simpson, E. M., Hynynen, K., & Aubert, I. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.10.001 · Cited by 30

Human cell surface-AAV interactomes identify LRP6 as blood-brain barrier transcytosis receptor and immune cytokine IL3 as AAV9 binder

Shay, T. F., Jang, S., Brittain, T. J., Chen, X., Walker, B., Tebbutt, C., Fan, Y., Wolfe, D. A., Arokiaraj, C. M., Sullivan, E. E., et al. (2024) · Nature Communications · doi:10.1038/s41467-024-52149-0 · Cited by 28

Systemic administration of AAV-Slc25a46 mitigates mitochondrial neuropathy in Slc25a46−/− mice

Yang, L., Slone, J. D., Li, Z., Lou, X., Hu, Y., Queme, L. F., Jankowski, M. P., & Huang, T. (2020) · Human Molecular Genetics · doi:10.1093/hmg/ddz277 · Cited by 27

Directed evolution of AAV accounting for long-term and enhanced transduction of cardiovascular endothelial cells in vivo

Liu, Y. B., Xu, B., Chen, Y., Yuan, X., Liu, J., Liu, T., Du, G. Z., Jiang, W., Yang, Y., Zhu, Y. F., et al. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.05.015 · Cited by 25

LY6S, a New IFN-Inducible Human Member of the Ly6a Subfamily Expressed by Spleen Cells and Associated with Inflammation and Viral Resistance

Shmerling, M., Chalik, M., Smorodinsky, N. I., Meeker, A. K., Roy, S., Sagi‐Assif, O., Meshel, T., Danilevsky, A., Shomron, N., Levinger, S., et al. (2022) · ImmunoHorizons · doi:10.4049/immunohorizons.2200018 · Cited by 23

Ancestral Library Identifies Conserved Reprogrammable Liver Motif on AAV Capsid

Zinn, E. M., Unzu, C., Schmit, P. F., Turunen, H., Zabaleta, N., Sanmiguel, J. C., Fieldsend, A., Bhatt, U., Diop, C. T., Merkel, E., et al. (2021) · Cell Reports Medicine · doi:10.1016/j.xcrm.2022.100803 · Cited by 28

A blood-brain barrier-penetrating AAV2 mutant created by a brain microvasculature endothelial cell-targeted AAV2 variant

Kawabata, H., Konno, A., Matsuzaki, Y., & Hirai, H. (2023) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2023.02.016 · Cited by 24

Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann–Pick disease type C1 mice

Davidson, C. D., Gibson, A. L., Gu, T., Baxter, L. L., Deverman, B. E., Beadle, K., Incao, A. A., Rodriguez‐Gil, J. L., Fujiwara, H., Jiang, X., et al. (2021) · Life Science Alliance · doi:10.26508/lsa.202101040 · Cited by 25

AAV-vector based gene therapy for mitochondrial disease: progress and future perspectives

Hanaford, A. R., Cho, Y., & Nakai, H. (2022) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-022-02324-7 · Cited by 25

Establishment of mouse model of inherited PIGO deficiency and therapeutic potential of AAV-based gene therapy

Kuwayama, R., Suzuki, K., Nakamura, J., Aizawa, E., Yoshioka, Y., Ikawa, M., Nabatame, S., Inoue, K., Shimmyo, Y., Ozono, K., et al. (2022) · Nature Communications · doi:10.1038/s41467-022-30847-x · Cited by 23

Apolipoprotein E, low-density lipoprotein receptor, and immune cells control blood-brain barrier penetration by AAV-PHP.eB in mice

Xie, B., Wang, X., Pan, Y., Jiang, G., Feng, J., & Lin, Y. (2020) · Theranostics · doi:10.7150/thno.46992 · Cited by 23

Enzyme Replacement Therapy for Succinic Semialdehyde Dehydrogenase Deficiency: Relevance in γ-Aminobutyric Acid Plasticity

Lee, H. H. C., Pearl, P. L., & Rotenberg, A. (2021) · Journal of Child Neurology · doi:10.1177/0883073821993000 · Cited by 22

Genetic Constructs for the Control of Astrocytes’ Activity

Borodinova, A. A., Balaban, P. M., Борисович, Б. И., Salmina, A. B., & Власова, О. Л. (2021) · Cells · doi:10.3390/cells10071600 · Cited by 22

Bioluminescence imaging in mice with synthetic luciferin analogues

Ji, X., Adams, S. T., & Miller, S. C. (2020) · Methods in enzymology on CD-ROM/Methods in enzymology · doi:10.1016/bs.mie.2020.04.033 · Cited by 19

Applications of adeno-associated virus vector-mediated gene delivery for neurodegenerative diseases and psychiatric diseases: Progress, advances, and challenges

Abulimiti, A., Lai, M. S., & Chang, R. C. (2021) · Mechanisms of Ageing and Development · doi:10.1016/j.mad.2021.111549 · Cited by 23

Engineering novel adeno-associated viruses (AAVs) for improved delivery in the nervous system

Carneiro, A. D., & Schaffer, D. V. (2024) · Current Opinion in Chemical Biology · doi:10.1016/j.cbpa.2024.102532 · Cited by 22

Fast, accurate ranking of engineered proteins by target-binding propensity using structure modeling

Ding, X., Chen, X., Sullivan, E. E., Shay, T. F., & Gradinaru, V. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.04.003 · Cited by 20

Small Alphaherpesvirus Latency-Associated Promoters Drive Efficient and Long-Term Transgene Expression in the CNS

Maturana, C. J., Verpeut, J. L., Pisano, T. J., Dhanerawala, Z. M., Esteves, A. D., Enquist, L. W., & Engel, E. A. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.04.004 · Cited by 19

Lynx1 prototoxins: critical accessory proteins of neuronal nicotinic acetylcholine receptors

Miwa, J. M. (2020) · Current Opinion in Pharmacology · doi:10.1016/j.coph.2020.09.016 · Cited by 20

Use of CRISPR/Cas9-mediated disruption of CNS cell type genes to profile transduction of AAV by neonatal intracerebroventricular delivery in mice

Torregrosa, T., Lehman, S., Hana, S., Marsh, G., Xu, S., Koszka, K., Mastrangelo, N., McCampbell, A., Henderson, C. E., & Lo, S. (2021) · Gene Therapy · doi:10.1038/s41434-021-00223-3 · Cited by 24

Next-Generation Gene Therapy for Parkinson’s Disease Using Engineered Viral Vectors

Björklund, T., & Davidsson, M. (2021) · Journal of Parkinson s Disease · doi:10.3233/jpd-212674 · Cited by 22

Complete Correction of Brain and Spinal Cord Pathology in Metachromatic Leukodystrophy Mice

Audouard, E., Oger, V., Meha, B., Cartier, N., Sevin, C., & Piguet, F. (2021) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2021.677895 · Cited by 20

Treatment of adult metachromatic leukodystrophy model mice using intrathecal administration of type 9 AAV vector encoding arylsulfatase A

Miyake, N., Miyake, K., Sakai, A., Yamamoto, M., Suzuki, H., & Shimada, T. (2021) · Scientific Reports · doi:10.1038/s41598-021-99979-2 · Cited by 18

CHARACTERIZATION OF THE AAV-PHP.B CAPSID AND ITS INTERACTION WITH THE LY6A RECEPTOR: IMPLICATIONS FOR RECEPTOR-TARGETED VECTOR ENGINEERING

Martino, R. (2022) · ScholarlyCommons (University of Pennsylvania)

Ly6a Differential Expression in Blood–Brain Barrier Is Responsible for Strain Specific Central Nervous System Transduction Profile of AAV-PHP.B

Batista, A. R., King, O. D., Reardon, C. P., Davis, C., Shankaracharya, Philip, V. M., Gray‐Edwards, H. L., D, N. A. M., Lutz, C. M., Landers, J. E., et al. (2019) · Human Gene Therapy · doi:10.1089/hum.2019.186 · Cited by 98

In This Issue

(2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.04.013

The GPI-Linked Protein LY6A Drives AAV-PHP.B Transport across the Blood-Brain Barrier

Hordeaux, J., Yuan, Y., Clark, P. M., Wang, Q., Martino, R. A., Sims, J. J., Bell, P., Raymond, A., Stanford, W. L., & Wilson, J. M. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.02.013 · Cited by 217

Neurotropic Properties of AAV-PHP.B Are Shared among Diverse Inbred Strains of Mice

Matsuzaki, Y., Tanaka, M., Hakoda, S., Masuda, T., Miyata, R., Konno, A., & Hirai, H. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.02.016 · Cited by 48