Immune-orthogonal orthologues of AAV capsids and of Cas9 circumvent the immune response to the administration of gene therapy

Multifunctional CRISPR-Cas9 with engineered immunosilenced human T cell epitopes

(2019) Shayesteh R. Ferdosi et al.   Nature Communications

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

In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation

(2018) Ana M. Moreno et al.   MOLECULAR THERAPY

Prevalence of Pre-existing Antibodies to CRISPR-Associated Nuclease Cas9 in the USA Population

(2018) Vijaya L. Simhadri et al.   Molecular Therapy-Methods & Clinical Development

High prevalence of Streptococcus pyogenes Cas9-reactive T cells within the adult human population

(2018) Dimitrios L. Wagner et al.   NATURE MEDICINE

Impact of AAV Capsid-Specific T-Cell Responses on Design and Outcome of Clinical Gene Transfer Trials with Recombinant Adeno-Associated Viral Vectors: An Evolving Controversy

(2017) Hildegund C.J. Ertl et al.   HUMAN GENE THERAPY

Regulatory and Exhausted T Cell Responses to AAV Capsid

(2017) Gwladys Gernoux et al.   HUMAN GENE THERAPY

Distinct T helper cell dependence of memory B-cell proliferation versus plasma cell differentiation

(2017) Franziska Zabel et al.   IMMUNOLOGY

Successful Repeated Hepatic Gene Delivery in Mice and Non-human Primates Achieved by Sequential Administration of AAV5 ch and AAV1

(2017) Anna Majowicz et al.   MOLECULAR THERAPY

Diversity and evolution of class 2 CRISPR–Cas systems

(2017) Sergey Shmakov et al.   NATURE REVIEWS MICROBIOLOGY

Immunity to CRISPR Cas9 and Cas12a therapeutics

(2017) Wei Leong Chew   Wiley Interdisciplinary Reviews-Systems Biology and Medicine

Therapeutic genome engineering via CRISPR-Cas systems

(2017) Ana M. Moreno et al.   Wiley Interdisciplinary Reviews-Systems Biology and Medicine

Inheritable Silencing of Endogenous Genes by Hit-and-Run Targeted Epigenetic Editing

(2016) Angelo Amabile et al.   CELL

Surveying the Delivery Methods of CRISPR/Cas9 for ex vivo Mammalian Cell Engineering

(2016) William J. Kelton et al.   CHIMIA

New CRISPR–Cas systems from uncultivated microbes

(2016) David Burstein et al.   NATURE

Analyzing CRISPR genome-editing experiments with CRISPResso

(2016) Luca Pinello et al.   NATURE BIOTECHNOLOGY

A multifunctional AAV–CRISPR–Cas9 and its host response

(2016) Wei Leong Chew et al.   NATURE METHODS

Sustained antigen availability during germinal center initiation enhances antibody responses to vaccination

(2016) Hok Hei Tam et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A large fraction of HLA class I ligands are proteasome-generated spliced peptides

(2016) Juliane Liepe et al.   SCIENCE

Direct measurement of T cell receptor affinity and sequence from naïve antiviral T cells

(2016) Shu-Qi Zhang et al.   Science Translational Medicine

AAV Natural Infection Induces Broad Cross-Neutralizing Antibody Responses to Multiple AAV Serotypes in Chimpanzees

(2016) Roberto Calcedo et al.   Human Gene Therapy Clinical Development

Viral Vectors for Gene Therapy: Translational and Clinical Outlook

(2015) Melissa A. Kotterman et al.   Annual Review of Biomedical Engineering

Gapped sequence alignment using artificial neural networks: application to the MHC class I system

(2015) Massimo Andreatta et al.   BIOINFORMATICS

Protein deimmunization via structure-based design enables efficient epitope deletion at high mutational loads

(2015) Regina S. Salvat et al.   BIOTECHNOLOGY AND BIOENGINEERING

Early Interaction of Adeno-Associated Virus Serotype 8 Vector with the Host Immune System Following Intramuscular Delivery Results in Weak but Detectable Lymphocyte and Dendritic Cell Transduction

(2015) Gwladys Gernoux et al.   HUMAN GENE THERAPY

Accurate pan-specific prediction of peptide-MHC class II binding affinity with improved binding core identification

(2015) Massimo Andreatta et al.   IMMUNOGENETICS

Discovery and Functional Characterization of Diverse Class 2 CRISPR-Cas Systems

(2015) Sergey Shmakov et al.   MOLECULAR CELL

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

An updated evolutionary classification of CRISPR–Cas systems

(2015) Kira S. Makarova et al.   NATURE REVIEWS MICROBIOLOGY

Development of an intein-mediated split–Cas9 system for gene therapy

(2015) Dong-Jiunn Jeffery Truong et al.   NUCLEIC ACIDS RESEARCH

In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector

(2015) Eric Zinn et al.   Cell Reports

Development and Applications of CRISPR-Cas9 for Genome Engineering

(2014) Patrick D. Hsu et al.   CELL

Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing

(2014) Qiurong Ding et al.   CIRCULATION RESEARCH

The immune epitope database (IEDB) 3.0

(2014) Randi Vita et al.   NUCLEIC ACIDS RESEARCH

Classification and evolution of type II CRISPR-Cas systems

(2014) Krzysztof Chylinski et al.   NUCLEIC ACIDS RESEARCH

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

(2013) F. Mingozzi et al.   BLOOD

T-cell dependent immunogenicity of protein therapeutics: Preclinical assessment and mitigation

(2013) Vibha Jawa et al.   CLINICAL IMMUNOLOGY

In vivo assessment of specific cytotoxic T lymphocyte killing

(2013) T. Clemente et al.   METHODS

Adeno-associated Virus-mediated Rescue of Neonatal Lethality in Argininosuccinate Synthetase-deficient Mice

(2013) Cindy Y Kok et al.   MOLECULAR THERAPY

Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease

(2013) Seung Woo Cho et al.   NATURE BIOTECHNOLOGY

Cas9 as a versatile tool for engineering biology

(2013) Prashant Mali et al.   NATURE METHODS

Challenges and approaches for the development of safer immunomodulatory biologics

(2013) Jean G. Sathish et al.   NATURE REVIEWS DRUG DISCOVERY

Phylogeny of Cas9 determines functional exchangeability of dual-RNA and Cas9 among orthologous type II CRISPR-Cas systems

(2013) Ines Fonfara et al.   NUCLEIC ACIDS RESEARCH

Genetic measurement of memory B-cell recall using antibody repertoire sequencing

(2013) C. Vollmers et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Exploring the MHC-peptide matrix of central tolerance in the human thymus

(2013) Eleni Adamopoulou et al.   Nature Communications

RNA-programmed genome editing in human cells

(2013) Martin Jinek et al.   eLife

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

(2012) F Mingozzi et al.   GENE THERAPY

Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates

(2012) Carmen Unzu et al.   Journal of Translational Medicine

Pharmacological Modulation of Humoral Immunity in a Nonhuman Primate Model of AAV Gene Transfer for Hemophilia B

(2012) Federico Mingozzi et al.   MOLECULAR THERAPY

Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria

(2012) G. Gasiunas et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine

(2011) J H McIntosh et al.   GENE THERAPY

Examining the cross-reactivity and neutralization mechanisms of a panel of mAbs against adeno-associated virus serotypes 1 and 5

(2011) C. E. Harbison et al.   JOURNAL OF GENERAL VIROLOGY

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

(2011) Federico Mingozzi et al.   NATURE REVIEWS GENETICS

PEGylation of therapeutic proteins

(2010) Simona Jevševar et al.   Biotechnology Journal

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

The immunogenicity of humanized and fully human antibodies

(2010) Fiona A. Harding et al.   mAbs

The Complex and Evolving Story of T cell Activation to AAV Vector-encoded Transgene Products

(2010) Lauren E Mays et al.   MOLECULAR THERAPY

The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice

(2009) Jiangao Zhu et al.   JOURNAL OF CLINICAL INVESTIGATION

The Impact of PEGylation on Biological Therapies

(2008) Francesco M Veronese et al.   BIODRUGS

Predicting linear B‐cell epitopes using string kernels

(2008) Yasser EL‐Manzalawy et al.   JOURNAL OF MOLECULAR RECOGNITION