Interspecies Scaling of Transgene Products for Viral Vector Gene Therapies: Method Assessment Using Data from Eleven Viral Vectors

Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy

(2023) Caroline Le Guiner et al.   Molecular Therapy-Methods & Clinical Development

The Perspective of DMPK on Recombinant Adeno-Associated Virus-Based Gene Therapy: Past Learning, Current Support, and Future Contribution

(2022) Nancy Chen et al.   AAPS Journal

Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A

(2022) Sylvia Fong et al.   NATURE MEDICINE

Interspecies normalization of dose‐response relationship for adeno‐associated virus‐mediated haemophilia gene therapy—Application to human dose prediction

(2022) Peng Zou   BRITISH JOURNAL OF CLINICAL PHARMACOLOGY

Clinical Pharmacology Considerations on Recombinant Adeno‐Associated Virus–Based Gene Therapy

(2022) Kefeng Sun et al.   JOURNAL OF CLINICAL PHARMACOLOGY

First-in-Patient Dose Prediction for Adeno-Associated Virus-Mediated Hemophilia Gene Therapy Using Allometric Scaling

(2022) Peng Zou   Molecular Pharmaceutics

A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2

(2022) Kevin M. Flanigan et al.   Molecular Therapy-Methods & Clinical Development

Anatomical and ontogenetic influences on muscle density

(2021) Kaitlyn C. Leonard et al.   Scientific Reports

Duchenne muscular dystrophy

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

Rational Clinical Dose Selection of Adeno‐Associated Virus‐Mediated Gene Therapy Based on Allometric Principles

(2021) Fei Tang et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Current and Next Steps Toward Prediction of Human Dose for Gene Therapy Using Translational Dose‐Response Studies

(2021) Sergey Aksenov et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Repeated systemic dosing of AAV vectors in immunocompetent mice after blockade of T-cell costimulatory pathways

(2021) Chen Zhong et al.   HUMAN GENE THERAPY

Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans

(2020) Gwladys Gernoux et al.   MOLECULAR THERAPY

Hemophilia A and B: molecular and clinical similarities and differences

(2019) Giancarlo Castaman et al.   HAEMATOLOGICA

Muscle Mass in Children and Adolescents: Proposed Equations and Reference Values for Assessment

(2019) Marco A. Cossio Bolaños et al.   Frontiers in Endocrinology

Update on clinical gene therapy for hemophilia

(2018) George Q. Perrin et al.   BLOOD

The translation of age-related body composition findings from rodents to humans

(2018) Lindsay E. Pappas et al.   EUROPEAN JOURNAL OF CLINICAL NUTRITION

Emerging Strategies in the Treatment of Duchenne Muscular Dystrophy

(2018) Perry B. Shieh   Neurotherapeutics

Contemporary Animal Models For Human Gene Therapy Applications

(2015) Chitra Gopinath et al.   CURRENT GENE THERAPY

Adeno-associated virus structural biology as a tool in vector development

(2013) Lauren M Drouin et al.   Future Virology

Phase I Trial of Intravesical Recombinant Adenovirus Mediated Interferon-α2b Formulated in Syn3 for Bacillus Calmette-Guérin Failures in Nonmuscle Invasive Bladder Cancer

(2013) Colin P.N. Dinney et al.   JOURNAL OF UROLOGY

Toxicity and exposure of an adenovirus containing human interferon alpha-2b following intracystic administration in cynomolgus monkeys

(2011) R W Veneziale et al.   GENE THERAPY

Clinical severity of haemophilia A: does the classification of the 1950s still stand?

(2011) I. E. M. Den UIJL et al.   HAEMOPHILIA

Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α1-Antitrypsin: Interim Results

(2011) Terence R. Flotte et al.   HUMAN GENE THERAPY

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

(2009) M. L. Brantly et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Effect of breed on body composition and comparison between various methods to estimate body composition in dogs

(2009) I. Jeusette et al.   RESEARCH IN VETERINARY SCIENCE