Development of Gene Therapy Vectors: Remaining Challenges

Emerging patent landscape for non-viral vectors used for gene therapy

(2020) Virginia Picanço-Castro et al.   NATURE BIOTECHNOLOGY

Capsid-specific removal of circulating antibodies to adeno-associated virus vectors

(2020) Berangere Bertin et al.   Scientific Reports

Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B

(2020) Lindsey A. George et al.   MOLECULAR THERAPY

Timing of Intensive Immunosuppression Impacts Risk of Transgene Antibodies after AAV Gene Therapy in Nonhuman Primates

(2020) Benjamin J. Samelson-Jones et al.   Molecular Therapy-Methods & Clinical Development

CRISPR activation enhances in vitro potency of AAV vectors driven by tissue-specific promoters

(2019) Devin S. McDougald et al.   Molecular Therapy-Methods & Clinical Development

Detection of Biologically Relevant Low-Titer Neutralizing Antibodies Against Adeno-Associated Virus Require Sensitive In Vitro Assays

(2019) Anita Kruzik et al.   Human Gene Therapy Methods

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

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

Clinical translation of gene medicine

(2019) Fazhan Wang et al.   JOURNAL OF GENE MEDICINE

Clinical Considerations for Capsid Choice in the Development of Liver-Targeted AAV-Based Gene Transfer

(2019) Steven Pipe et al.   Molecular Therapy-Methods & Clinical Development

Adeno-associated virus-mediated expression of human butyrylcholinesterase to treat organophosphate poisoning

(2019) Vibhor Gupta et al.   PLoS One

Poly (amidoamine) (PAMAM) dendrimer mediated delivery of drug and pDNA/siRNA for cancer therapy

(2018) Jun Li et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

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

An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs

(2018) Junjiang Sun et al.   Molecular Therapy-Methods & Clinical Development

Comparative analyses of adeno-associated viral vector serotypes 1, 2, 5, 8 and 9 in marmoset, mouse and macaque cerebral cortex

(2015) Akiya Watakabe et al.   NEUROSCIENCE RESEARCH

Adeno-Associated Virus 9-Mediated Airway Expression of Antibody Protects Old and Immunodeficient Mice against Influenza Virus

(2014) Virginie S. Adam et al.   Clinical and Vaccine Immunology

AAV Empty Capsids: For Better or for Worse?

(2014) J Fraser Wright   MOLECULAR THERAPY

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

(2013) F. Mingozzi et al.   BLOOD

Vectored Expression of the Broadly Neutralizing Antibody FI6 in Mouse Airway Provides Partial Protection against a New Avian Influenza A Virus, H7N9

(2013) Maria P. Limberis et al.   Clinical and Vaccine Immunology

Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys

(2013) F. Mingozzi et al.   Science Translational Medicine

Advances in Liver-Directed Gene Therapy for Hepatocellular Carcinoma by Non-Viral Delivery Systems

(2012) Buyun Ding et al.   CURRENT GENE THERAPY

Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue

(2012) F Mingozzi et al.   GENE THERAPY

Development and validation of cell-based assays for the detection of neutralizing antibodies to drug products: a practical approach

(2012) Pierre Jolicoeur et al.   Bioanalysis

The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver

(2011) A. T. Martino et al.   BLOOD

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

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

Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia

(2011) C Li et al.   GENE THERAPY

Impact of Pre-Existing Immunity on Gene Transfer to Nonhuman Primate Liver with Adeno-Associated Virus 8 Vectors

(2011) Lili Wang et al.   HUMAN GENE THERAPY

Systemic Administration of AAV8-α-galactosidase A Induces Humoral Tolerance in Nonhuman Primates Despite Low Hepatic Expression

(2011) Jennifer B Nietupski et al.   MOLECULAR THERAPY

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

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

Efficient Serotype-Dependent Release of Functional Vector into the Culture Medium During Adeno-Associated Virus Manufacturing

(2010) Luk H. Vandenberghe et al.   HUMAN GENE THERAPY

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

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

(2009) Roberto Calcedo et al.   JOURNAL OF INFECTIOUS DISEASES

Characterization of the transgene expression generated by branched and linear polyethylenimine-plasmid DNA nanoparticles in vitro and after intraperitoneal injection in vivo

(2008) Janjira Intra et al.   JOURNAL OF CONTROLLED RELEASE

Diverse IgG subclass responses to adeno-associated virus infection and vector administration

(2008) Samuel L. Murphy et al.   JOURNAL OF MEDICAL VIROLOGY

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY