Rational engineering of a functional CpG-free ITR for AAV gene therapy

Synthetic Biology: Emerging Concepts to Design and Advance Adeno‐Associated Viral Vectors for Gene Therapy

(2021) Hanna J. Wagner et al.   Advanced Science

Duchenne muscular dystrophy

(2021) Dongsheng Duan et al.   Nature Reviews Disease Primers

Effect of CpG Depletion of Vector Genome on CD8+ T Cell Responses in AAV Gene Therapy

(2021) Thais B. Bertolini et al.   Frontiers in Immunology

ITRs: The Terminal Frontier

(2020) Phillip W.L. Tai   HUMAN GENE THERAPY

Adeno-Associated Virus Serotype-Specific Inverted Terminal Repeat Sequence Role in Vector Transgene Expression

(2020) Lauriel F. Earley et al.   HUMAN GENE THERAPY

The Unusual Properties of the AAV Inverted Terminal Repeat

(2020) Kenneth I. Berns   HUMAN GENE THERAPY

Codon Modification and PAMPs in Clinical AAV Vectors: The Tortoise or the Hare?

(2020) J. Fraser Wright   MOLECULAR THERAPY

Engineering adeno-associated virus vectors for gene therapy

(2020) Chengwen Li et al.   NATURE REVIEWS GENETICS

Voretigene neparvovec-rzyl for treatment of RPE65-mediated inherited retinal diseases: a model for ocular gene therapy development

(2020) Thomas A. Ciulla et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

BAX 335 hemophilia B gene therapy clinical trial results - potential impact of CpG sequences on gene expression

(2020) Barbara A Konkle et al.   BLOOD

AAV-Genome Population Sequencing of Vectors Packaging CRISPR Components Reveals Design-Influenced Heterogeneity

(2020) Ngoc Tam Tran et al.   Molecular Therapy-Methods & Clinical Development

Adeno-associated virus vector as a platform for gene therapy delivery

(2019) Dan Wang et al.   NATURE REVIEWS DRUG DISCOVERY

Onasemnogene Abeparvovec: First Global Approval

(2019) Sheridan M. Hoy   DRUGS

Gene Therapy

(2019) Katherine A. High et al.   NEW ENGLAND JOURNAL OF MEDICINE

Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

(2019) Allison M. Keeler et al.   Annual Review of Virology

A User's Guide to the Inverted Terminal Repeats of Adeno-Associated Virus

(2019) Patrick Wilmott et al.   Human Gene Therapy Methods

Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy

(2018) Dongsheng Duan   MOLECULAR THERAPY

Adeno-associated Virus Genome Population Sequencing Achieves Full Vector Genome Resolution and Reveals Human-Vector Chimeras

(2018) Phillip W.L. Tai et al.   Molecular Therapy-Methods & Clinical Development

Understanding capsid assembly and genome packaging for adeno-associated viruses

(2017) Antonette Bennett et al.   Future Virology

Impact of Inverted Terminal Repeat Integrity on rAAV8 Production Using the Baculovirus/Sf9 Cells System

(2017) Adrien Savy et al.   Human Gene Therapy Methods

A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy

(2017) Chady H. Hakim et al.   Molecular Therapy-Methods & Clinical Development

Deletion of the B-B’ and C-C’ regions of inverted terminal repeats reduces rAAV productivity but increases transgene expression

(2017) Qingzhang Zhou et al.   Scientific Reports

Adeno-Associated Virus at 50: A Golden Anniversary of Discovery, Research, and Gene Therapy Success—A Personal Perspective

(2015) Eric Hastie et al.   HUMAN GENE THERAPY

AAV's Golden Jubilee

(2015) Nicholas Muzyczka et al.   MOLECULAR THERAPY

Enhanced Transgene Expression from Recombinant Single-Stranded D-Sequence-Substituted Adeno-Associated Virus Vectors in Human Cell LinesIn Vitroand in Murine HepatocytesIn Vivo

(2014) Chen Ling et al.   JOURNAL OF VIROLOGY

Engineering adeno-associated viruses for clinical gene therapy

(2014) Melissa A. Kotterman et al.   NATURE REVIEWS GENETICS

Optimization of AAV expression cassettes to improve packaging capacity and transgene expression in neurons

(2014) Jun-Hyeok Choi et al.   Molecular Brain

AAV-Mediated Gene Therapy for Research and Therapeutic Purposes

(2014) R. Jude Samulski et al.   Annual Review of Virology

CpG-depleted adeno-associated virus vectors evade immune detection

(2013) Susan M. Faust et al.   JOURNAL OF CLINICAL INVESTIGATION

Microdystrophin Ameliorates Muscular Dystrophy in the Canine Model of Duchenne Muscular Dystrophy

(2013) Jin-Hong Shin et al.   MOLECULAR THERAPY

Fiji: an open-source platform for biological-image analysis

(2012) Johannes Schindelin et al.   NATURE METHODS

Integrative genomics viewer

(2011) James T Robinson et al.   NATURE BIOTECHNOLOGY

Genotyping mdx, mdx3cv, and mdx4cv mice by primer competition polymerase chain reaction

(2010) Jin-Hong Shin et al.   MUSCLE & NERVE

Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy

(2009) Yi Lai et al.   JOURNAL OF CLINICAL INVESTIGATION

Evidence for the Failure of Adeno-associated Virus Serotype 5 to Package a Viral Genome ≥8.2 kb

(2009) Yi Lai et al.   MOLECULAR THERAPY

Adeno-associated Virus of a Single-polarity DNA Genome Is Capable of Transduction In Vivo

(2008) Xiaohuai Zhou et al.   MOLECULAR THERAPY

High-efficiency Transduction of the Mouse Retina by Tyrosine-mutant AAV Serotype Vectors

(2008) Hilda Petrs-Silva et al.   MOLECULAR THERAPY

CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression

(2008) Stephen C Hyde et al.   NATURE BIOTECHNOLOGY

Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses

(2008) L. Zhong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Single-polarity Recombinant Adeno-associated Virus 2 Vector-mediated Transgene Expression In Vitro and In Vivo: Mechanism of Transduction

(2007) Li Zhong et al.   MOLECULAR THERAPY

A Hybrid Vector System Expands Adeno-associated Viral Vector Packaging Capacity in a Transgene-independent Manner

(2007) Arkasubhra Ghosh et al.   MOLECULAR THERAPY