Selection of appropriate non-clinical animal models to ensure translatability of novel AAV-gene therapies to the clinic

Why gene therapies must go virus-free

(2023) Cormac Sheridan   NATURE BIOTECHNOLOGY

Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution

(2023) Marti Cabanes-Creus et al.   Molecular Therapy-Methods & Clinical Development

Importance of non-human primates as model system for gene therapy development in ophthalmology

(2022) Fabian Wozar et al.   KLINISCHE MONATSBLATTER FUR AUGENHEILKUNDE

Gene Therapy Advances: A Meta-Analysis of AAV Usage in Clinical Settings

(2022) Hau Kiu Edna Au et al.   Frontiers in Medicine

Addressing high dose AAV toxicity – ‘one and done’ or ‘slower and lower’?

(2022) Takashi Kei Kishimoto et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders

(2022) Francesca Tucci et al.   Nature Communications

Immunogenicity and toxicity of AAV gene therapy

(2022) Hildegund C. J. Ertl   Frontiers in Immunology

Optimized AAV Vectors for TMC1 Gene Therapy in a Humanized Mouse Model of DFNB7/11

(2022) Irina Marcovich et al.   Biomolecules

The clinical landscape for AAV gene therapies

(2021) Dmitry A. Kuzmin et al.   NATURE REVIEWS DRUG DISCOVERY

Evolving AAV-delivered therapeutics towards ultimate cures

(2021) Xiangjun He et al.   JOURNAL OF MOLECULAR MEDICINE-JMM

CAR-T cell therapy: current limitations and potential strategies

(2021) Robert C. Sterner et al.   Blood Cancer Journal

T Cell-Mediated Immune Responses to AAV and AAV Vectors

(2021) Hildegund C. J. Ertl   Frontiers in Immunology

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

(2021) Chen Zhong et al.   HUMAN GENE THERAPY

Huntingtin and the Synapse

(2021) Jessica C. Barron et al.   Frontiers in Cellular Neuroscience

Precise CAG repeat contraction in a Huntington’s Disease mouse model is enabled by gene editing with SpCas9-NG

(2021) Seiya Oura et al.   Communications Biology

Fourth Boy Dies in Clinical Trial of Astellas' AT132

(2021) Alex Philippidis   HUMAN GENE THERAPY

Species selection for nonclinical safety assessment of drug candidates: Examples of current industry practice

(2021) Rostam Namdari et al.   REGULATORY TOXICOLOGY AND PHARMACOLOGY

Utility of microminipigs for evaluating liver-mediated gene expression in the presence of neutralizing antibody against vector capsid

(2020) Ryota Watano et al.   GENE THERAPY

AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer

(2020) Helena Costa Verdera et al.   MOLECULAR THERAPY

Focus shifts to antibody cocktails for COVID-19 cytokine storm

(2020) Charlotte Harrison   NATURE BIOTECHNOLOGY

Towards Clinical Implementation of Adeno-Associated Virus (AAV) Vectors for Cancer Gene Therapy: Current Status and Future Perspectives

(2020) Ulrich T. Hacker et al.   Cancers

Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology

(2020) Juliette Hordeaux et al.   HUMAN GENE THERAPY

After Third Death, Audentes' AT132 Remains on Clinical Hold

(2020) Alex Philippidis   HUMAN GENE THERAPY

Huntington disease: new insights into molecular pathogenesis and therapeutic opportunities

(2020) Sarah J. Tabrizi et al.   Nature Reviews Neurology

Advances in selecting appropriate non-rodent species for regulatory toxicology research: Policy, ethical, and experimental considerations

(2020) Yong-Wook Son et al.   REGULATORY TOXICOLOGY AND PHARMACOLOGY

Gene therapy for monogenic inherited disorders—opportunities and challenges

(2020) Janbernd Kirschner et al.   Deutsches Arzteblatt International

Current Clinical Applications of In Vivo Gene Therapy with AAVs

(2020) Jerry R. Mendell et al.   MOLECULAR THERAPY

The once and future gene therapy

(2020) Karen Bulaklak et al.   Nature Communications

Predictivity/Translatability of Toxicities Observed in Nonclinical Toxicology Studies to Clinical Safety Outcomes in Drug Development: Case Examples

(2019) Nicola J. Stagg et al.   INTERNATIONAL JOURNAL OF TOXICOLOGY

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

(2018) Christian Hinderer et al.   HUMAN GENE THERAPY

AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington’s Disease Minipig Model

(2018) Melvin M. Evers et al.   MOLECULAR THERAPY

Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility

(2018) Jayesh A. Kulkarni et al.   Nucleic Acid Therapeutics

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

(2018) Pasqualina Colella et al.   Molecular Therapy-Methods & Clinical Development

Huntington's disease: a clinical review

(2017) P. McColgan et al.   EUROPEAN JOURNAL OF NEUROLOGY

Lipid Nanoparticle Systems for Enabling Gene Therapies

(2017) Pieter R. Cullis et al.   MOLECULAR THERAPY

After Glybera's withdrawal, what's next for gene therapy?

(2017) Melanie Senior   NATURE BIOTECHNOLOGY

Next-generation AAV vectors for clinical use: an ever-accelerating race

(2017) Jonas Weinmann et al.   VIRUS GENES

Persistent Expression of Dopamine-Synthesizing Enzymes 15 Years After Gene Transfer in a Primate Model of Parkinson's Disease

(2017) Yoshihide Sehara et al.   Human Gene Therapy Clinical Development

Current Progress in Therapeutic Gene Editing for Monogenic Diseases

(2016) Versha Prakash et al.   MOLECULAR THERAPY

Genotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy?

(2016) Randy J Chandler et al.   MOLECULAR THERAPY

In vivo tissue-tropism of adeno-associated viral vectors

(2016) Arun Srivastava   Current Opinion in Virology

E Pluribus Unum: 50 Years of Research, Millions of Viruses, and One Goal—Tailored Acceleration of AAV Evolution

(2015) Dirk Grimm et al.   MOLECULAR THERAPY

Basic Biology of Adeno-Associated Virus (AAV) Vectors Used in Gene Therapy

(2014) Balaji Balakrishnan et al.   CURRENT GENE THERAPY

Gene Therapies for Cancer: Strategies, Challenges and Successes

(2014) Swadesh K. Das et al.   JOURNAL OF CELLULAR PHYSIOLOGY

Engineering adeno-associated viruses for clinical gene therapy

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

Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse

(2014) Daniel J. Schuster et al.   Frontiers in Neuroanatomy

Treatment of Diabetes and Long-Term Survival After Insulin and Glucokinase Gene Therapy

(2013) D. Callejas et al.   DIABETES

The immune privilege of testis and gravid uterus: Same difference?

(2013) Petra Arck et al.   MOLECULAR AND CELLULAR ENDOCRINOLOGY

Pre-existing Anti–Adeno-Associated Virus Antibodies as a Challenge in AAV Gene Therapy

(2013) Vedell Louis Jeune et al.   Human Gene Therapy Methods

Lessons Learned from the Clinical Development and Market Authorization of Glybera

(2013) Laura M. Bryant et al.   Human Gene Therapy Clinical Development

Directed evolution of novel adeno-associated viruses for therapeutic gene delivery

(2012) M A Bartel et al.   GENE THERAPY

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

(2012) D Gaudet et al.   GENE THERAPY

The Minipig as Nonrodent Species in Toxicology—Where Are We Now?

(2012) Niels Christian Ganderup et al.   INTERNATIONAL JOURNAL OF TOXICOLOGY

rAAV-Mediated Tumorigenesis: Still Unresolved After an AAV Assault

(2012) Paul N Valdmanis et al.   MOLECULAR THERAPY

The privileged immunity of immune privileged organs: the case of the eye

(2012) Inbal Benhar et al.   Frontiers in Immunology

Adeno-Associated Virus Antibody Profiles in Newborns, Children, and Adolescents

(2011) Roberto Calcedo et al.   Clinical and Vaccine Immunology

Considerations Regarding Nonhuman Primate Use in Safety Assessment of Biopharmaceuticals

(2011) Lorrene A. Buckley et al.   INTERNATIONAL JOURNAL OF TOXICOLOGY

Enhancing the clinical potential of AAV vectors by capsid engineering to evade pre-existing immunity

(2011) Melissa Bartel   Frontiers in Microbiology

Assessing the potential for AAV vector genotoxicity in a murine model

(2010) H. Li et al.   BLOOD

Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors

(2010) Sylvie Boutin et al.   HUMAN GENE THERAPY

Regulatory acceptability of the minipig in the development of pharmaceuticals, chemicals and other products

(2010) Jan Willem van der Laan et al.   JOURNAL OF PHARMACOLOGICAL AND TOXICOLOGICAL METHODS

Host and Vector-dependent Effects on the Risk of Germline Transmission of AAV Vectors

(2009) Patricia Favaro et al.   MOLECULAR THERAPY

Adeno-associated virus integration: virus versus vector

(2008) R H Smith   GENE THERAPY