Cas9-directed immune tolerance in humans—a model to evaluate regulatory T cells in gene therapy?

Regulatory T Cell Development

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

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

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The promise and challenge of therapeutic genome editing

(2020) Jennifer A. Doudna   NATURE

CRISPR-engineered T cells in patients with refractory cancer

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Management of Neuroinflammatory Responses to AAV-Mediated Gene Therapies for Neurodegenerative Diseases

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CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors

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Regulatory cell therapy in kidney transplantation (The ONE Study): a harmonised design and analysis of seven non-randomised, single-arm, phase 1/2A trials

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AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9

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Safety and feasibility of CRISPR-edited T cells in patients with refractory non-small-cell lung cancer

(2020) You Lu et al.   NATURE MEDICINE

A Potent Branched-Tail Lipid Nanoparticle Enables Multiplexed mRNA Delivery and Gene Editing In Vivo

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Gene editing to induce FOXP3 expression in human CD4+ T cells leads to a stable regulatory phenotype and function

(2020) Yuchi Honaker et al.   Science Translational Medicine

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

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Regulatory T cells for minimising immune suppression in kidney transplantation: phase I/IIa clinical trial

(2020) Andy Roemhild et al.   BMJ-British Medical Journal

Regulatory T cells mediate specific suppression by depleting peptide–MHC class II from dendritic cells

(2019) Billur Akkaya et al.   NATURE IMMUNOLOGY

Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10

(2019) Morgan L. Maeder et al.   NATURE MEDICINE

SpCas9-expression by tumor cells can cause T cell-dependent tumor rejection in immunocompetent mice

(2019) Reham Ajina et al.   OncoImmunology

A New Class of Medicines through DNA Editing

(2019) Matthew H. Porteus   NEW ENGLAND JOURNAL OF MEDICINE

Identification of preexisting adaptive immunity to Cas9 proteins in humans

(2019) Carsten T. Charlesworth et al.   NATURE MEDICINE

CasX enzymes comprise a distinct family of RNA-guided genome editors

(2019) Jun-Jie Liu et al.   NATURE

Delivery Aspects of CRISPR/Cas for in Vivo Genome Editing

(2019) Danny Wilbie et al.   ACCOUNTS OF CHEMICAL RESEARCH

Multifunctional CRISPR-Cas9 with engineered immunosilenced human T cell epitopes

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

Therapeutically relevant engraftment of a CRISPR-Cas9–edited HSC-enriched population with HbF reactivation in nonhuman primates

(2019) Olivier Humbert et al.   Science Translational Medicine

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

(2019) Ana M. Moreno et al.   Nature Biomedical Engineering

Doxycycline-Dependent Self-Inactivation of CRISPR-Cas9 to Temporally Regulate On- and Off-Target Editing

(2019) Anju Kelkar et al.   MOLECULAR THERAPY

CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia

(2019) Lei Xu et al.   NEW ENGLAND JOURNAL OF MEDICINE

Antigen‐specific in vitro expansion of factor VIII‐specific regulatory T cells induces tolerance in hemophilia A mice

(2019) Bryn M. Smith et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Search-and-replace genome editing without double-strand breaks or donor DNA

(2019) Andrew V. Anzalone et al.   NATURE

Engineered materials for in vivo delivery of genome-editing machinery

(2019) Sheng Tong et al.   Nature Reviews Materials

Evolutionary classification of CRISPR–Cas systems: a burst of class 2 and derived variants

(2019) Kira S. Makarova et al.   NATURE REVIEWS MICROBIOLOGY

Cross-Reactive Donor-Specific CD8+ Tregs Efficiently Prevent Transplant Rejection

(2019) Elodie Picarda et al.   Cell Reports

Somatic Editing ofLdlrWith Adeno-Associated Viral-CRISPR Is an Efficient Tool for Atherosclerosis Research

(2018) Kelsey E. Jarrett et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

Gene therapy clinical trials worldwide to 2017: An update

(2018) Samantha L. Ginn et al.   JOURNAL OF GENE MEDICINE

A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

(2018) Jonathan D. Finn et al.   Cell Reports

In vitro–transcribed guide RNAs trigger an innate immune response via the RIG-I pathway

(2018) Beeke Wienert et al.   PLOS BIOLOGY

Gene Therapy With Regulatory T Cells: A Beneficial Alliance

(2018) Moanaro Biswas et al.   Frontiers in Immunology

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

c-MAF-dependent regulatory T cells mediate immunological tolerance to a gut pathobiont

(2018) Mo Xu et al.   NATURE

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

(2018) Christopher A. Vakulskas et al.   NATURE MEDICINE

Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

(2018) Leonela Amoasii et al.   SCIENCE

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

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

Regulatory T cells in the treatment of disease

(2018) Amir Sharabi et al.   NATURE REVIEWS DRUG DISCOVERY

Genome editing in primary cells and in vivo using viral-derived Nanoblades loaded with Cas9-sgRNA ribonucleoproteins

(2018) Philippe E. Mangeot et al.   Nature Communications

Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration

(2018) Amine Meliani et al.   Nature Communications

A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing

(2018) Ang Li et al.   Molecular Therapy-Methods & Clinical Development

Entering the Modern Era of Gene Therapy

(2018) Xavier M. Anguela et al.   Annual Review of Medicine

Oral Tolerance Induction in Hemophilia B Dogs Fed with Transplastomic Lettuce

(2017) Roland W. Herzog et al.   MOLECULAR THERAPY

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

(2017) Nicole M. Gaudelli et al.   NATURE

Efficient genome editing in the mouse brain by local delivery of engineered Cas9 ribonucleoprotein complexes

(2017) Brett T Staahl et al.   NATURE BIOTECHNOLOGY

Immunity to CRISPR Cas9 and Cas12a therapeutics

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

Hit and go CAS9 delivered through a lentiviral based self-limiting circuit

(2017) Gianluca Petris et al.   Nature Communications

Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape

(2017) Federico Mingozzi et al.   Annual Review of Virology

Effective Depletion of Pre-existing Anti-AAV Antibodies Requires Broad Immune Targeting

(2017) Victoria M. Velazquez et al.   Molecular Therapy-Methods & Clinical Development

Regulatory T Cell Specificity Directs Tolerance versus Allergy against Aeroantigens in Humans

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The Neisseria meningitidis CRISPR-Cas9 System Enables Specific Genome Editing in Mammalian Cells

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Streptococcus thermophilus CRISPR-Cas9 Systems Enable Specific Editing of the Human Genome

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A multifunctional AAV–CRISPR–Cas9 and its host response

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Plant-based oral tolerance to hemophilia therapy employs a complex immune regulatory response including LAP+CD4+ T cells

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High proportions of regulatory T cells in PBSC grafts predict improved survival after allogeneic haematopoietic SCT

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Adenovirus-Mediated Somatic Genome Editing ofPtenby CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses

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Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

Therapeutic genome editing: prospects and challenges

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Novel GMP-Compatible Protocol Employing an Allogeneic B Cell Bank for Clonal Expansion of Allospecific Natural Regulatory T Cells

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Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins

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The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

Ex vivo expanded autologous polyclonal regulatory T cells suppress inhibitor formation in hemophilia

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Clinical Grade Manufacturing of Human Alloantigen-Reactive Regulatory T Cells for Use in Transplantation

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

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Human Treg responses allow sustained recombinant adeno-associated virus–mediated transgene expression

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HCMV-specific T-cell Therapy

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RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

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

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

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Dystrophin Immunity in Duchenne's Muscular Dystrophy

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Human Antigen-Specific Regulatory T Cells Generated by T Cell Receptor Gene Transfer

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Hepatic Gene Transfer as a Means of Tolerance Induction to Transgene Products

(2009) Paul LoDuca et al.   CURRENT GENE THERAPY

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

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Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

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Conferring indirect allospecificity on CD4+CD25+ Tregs by TCR gene transfer favors transplantation tolerance in mice

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Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

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Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

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