Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges

Targeting herpes simplex virus with CRISPR–Cas9 cures herpetic stromal keratitis in mice

(2021) Di Yin et al.   NATURE BIOTECHNOLOGY

Identifying genome-wide off-target sites of CRISPR RNA–guided nucleases and deaminases with Digenome-seq

(2021) Daesik Kim et al.   Nature Protocols

In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA

(2021) Lukas Villiger et al.   Nature Biomedical Engineering

Engineered prime editors with PAM flexibility

(2021) Jiyeon Kweon et al.   MOLECULAR THERAPY

Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3

(2021) Min Qiu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Precise base editing for the in vivo study of developmental signaling and human pathologies in zebrafish

(2021) Marion Rosello et al.   eLife

Virus-like particles: preparation, immunogenicity and their roles as nanovaccines and drug nanocarriers

(2021) Saghi Nooraei et al.   JOURNAL OF NANOBIOTECHNOLOGY

Efficient ssODN-Mediated Targeting by Avoiding Cellular Inhibitory RNAs through Precomplexed CRISPR-Cas9/sgRNA Ribonucleoprotein

(2021) Akihiro Kagita et al.   Stem Cell Reports

Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease

(2021) Naoya Uchida et al.   Molecular Therapy-Methods & Clinical Development

Genome-wide programmable transcriptional memory by CRISPR-based epigenome editing

(2021) James K. Nuñez et al.   CELL

Co-encapsulation of Cas9 mRNA and guide RNA in polyplex micelles enables genome editing in mouse brain

(2021) Saed Abbasi et al.   JOURNAL OF CONTROLLED RELEASE

In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates

(2021) Kiran Musunuru et al.   NATURE

In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels

(2021) Tanja Rothgangl et al.   NATURE BIOTECHNOLOGY

Scaffold-mediated CRISPR-Cas9 delivery system for acute myeloid leukemia therapy

(2021) Tzu-Chieh Ho et al.   Science Advances

Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species

(2021) Mohammadsharif Tabebordbar et al.   CELL

Prime editing – an update on the field

(2021) Janine Scholefield et al.   GENE THERAPY

Lipid-Based Nanoparticles for Delivery of Vaccine Adjuvants and Antigens: Toward Multicomponent Vaccines

(2021) Despo Chatzikleanthous et al.   MOLECULAR PHARMACEUTICS

Detect-seq reveals out-of-protospacer editing and target-strand editing by cytosine base editors

(2021) Zhixin Lei et al.   NATURE METHODS

Transgenic mice for in vivo epigenome editing with CRISPR-based systems

(2021) Matthew P. Gemberling et al.   NATURE METHODS

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

(2021) Julian D. Gillmore et al.   NEW ENGLAND JOURNAL OF MEDICINE

Efficient precise in vivo base editing in adult dystrophic mice

(2021) Li Xu et al.   Nature Communications

Genetically blocking HPD via CRISPR-Cas9 protects against lethal liver injury in a pig model of tyrosinemia type I

(2021) Peng Gu et al.   Molecular Therapy-Methods & Clinical Development

In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges

(2021) Matthew Behr et al.   Acta Pharmaceutica Sinica B

Efficient Peptide-Mediated In Vitro Delivery of Cas9 RNP

(2021) Oskar Gustafsson et al.   Pharmaceutics

Low immunogenicity of LNP allows repeated administrations of CRISPR-Cas9 mRNA into skeletal muscle in mice

(2021) Eriya Kenjo et al.   Nature Communications

Integrating gene delivery and gene-editing technologies by adenoviral vector transfer of optimized CRISPR-Cas9 components

(2020) Ignazio Maggio et al.   GENE THERAPY

A primate view of gene expression

(2020) Linda Koch   NATURE REVIEWS GENETICS

CRISPR-engineered T cells in patients with refractory cancer

(2020) Edward A. Stadtmauer et al.   SCIENCE

Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in mice

(2020) Koji M. Nishiguchi et al.   Nature Communications

A mutation-independent CRISPR-Cas9–mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiency

(2020) Lili Wang et al.   Science Advances

Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system

(2020) Yu Zhang et al.   Science Advances

A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I

(2020) Li Ou et al.   MOLECULAR THERAPY

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

(2020) Ang Li et al.   MOLECULAR THERAPY

Correction of three prominent mutations in mouse and human models of Duchenne muscular dystrophy by single-cut genome editing

(2020) Yi-Li Min et al.   MOLECULAR THERAPY

A rationally engineered cytosine base editor retains high on-target activity while reducing both DNA and RNA off-target effects

(2020) Erwei Zuo et al.   NATURE METHODS

Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing

(2020) Qiang Cheng et al.   Nature Nanotechnology

Gene-editing pipeline takes off

(2020) Asher Mullard   NATURE REVIEWS DRUG DISCOVERY

Cytosine base editors with minimized unguided DNA and RNA off-target events and high on-target activity

(2020) Yi Yu et al.   Nature Communications

Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping

(2020) Peter Gee et al.   Nature Communications

Recent Advances in CRISPR/Cas9 Delivery Strategies

(2020) Bon Ham Yip   Biomolecules

Viral nanoparticles for drug delivery, imaging, immunotherapy, and theranostic applications

(2020) Young Hun Chung et al.   ADVANCED DRUG DELIVERY REVIEWS

Phase 1/2 study of COVID-19 RNA vaccine BNT162b1 in adults

(2020) Mark J. Mulligan et al.   NATURE

Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing

(2020) Tuo Wei et al.   Nature Communications

Germline CRISPR/Cas9-Mediated Gene Editing Prevents Vision Loss in a Novel Mouse Model of Aniridia

(2020) Seyedeh Zeinab Mirjalili Mohanna et al.   Molecular Therapy-Methods & Clinical Development

Non-viral Gene Disruption by CRISPR/Cas9 Delivery Using Cell-permeable and Protein-stabilizing 30Kc19 Protein

(2020) Yu Jin Kim et al.   BIOTECHNOLOGY AND BIOPROCESS ENGINEERING

Predicting the efficiency of prime editing guide RNAs in human cells

(2020) Hui Kwon Kim et al.   NATURE BIOTECHNOLOGY

Safety and Immunogenicity of Two RNA-Based Covid-19 Vaccine Candidates

(2020) Edward E. Walsh et al.   NEW ENGLAND JOURNAL OF MEDICINE

Prime editing for functional repair in patient-derived disease models

(2020) Imre F. Schene et al.   Nature Communications

Delivery Approaches for Therapeutic Genome Editing and Challenges

(2020) Ilayda Ates et al.   Genes

A Cas-embedding strategy for minimizing off-target effects of DNA base editors

(2020) Yajing Liu et al.   Nature Communications

Lipid nanoparticles loaded with ribonucleoprotein–oligonucleotide complexes synthesized using a microfluidic device exhibit robust genome editing and hepatitis B virus inhibition

(2020) Yuichi Suzuki et al.   JOURNAL OF CONTROLLED RELEASE

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

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

Genome-wide profiling of adenine base editor specificity by EndoV-seq

(2019) Puping Liang et al.   Nature Communications

Engineering of CRISPR-Cas12b for human genome editing

(2019) Jonathan Strecker et al.   Nature Communications

CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells

(2019) Yi-Li Min et al.   Science Advances

Engineering of high-precision base editors for site-specific single nucleotide replacement

(2019) Junjie Tan et al.   Nature Communications

Cytosine base editor generates substantial off-target single-nucleotide variants in mouse embryos

(2019) Erwei Zuo et al.   SCIENCE

Cytosine, but not adenine, base editors induce genome-wide off-target mutations in rice

(2019) Shuai Jin et al.   SCIENCE

Identification of preexisting adaptive immunity to Cas9 proteins in humans

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

Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy

(2019) Christopher E. Nelson et al.   NATURE MEDICINE

Genome-wide target specificity of CRISPR RNA-guided adenine base editors

(2019) Daesik Kim et al.   NATURE BIOTECHNOLOGY

Recent Advances in Cell Penetrating Peptide-Based Anticancer Therapies

(2019) Justine Habault et al.   MOLECULES

Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9

(2019) Calvin J. Stephens et al.   JOURNAL OF CONTROLLED RELEASE

Targeted Disruption of HLA Genes via CRISPR-Cas9 Generates iPSCs with Enhanced Immune Compatibility

(2019) Huaigeng Xu et al.   Cell Stem Cell

Delivery Aspects of CRISPR/Cas for in Vivo Genome Editing

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

Ultrasound microbubble-mediated CRISPR/Cas9 knockout of C-erbB-2 in HEC-1A cells

(2019) Junhong Cai et al.   JOURNAL OF INTERNATIONAL MEDICAL RESEARCH

Multifunctional CRISPR-Cas9 with engineered immunosilenced human T cell epitopes

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

Delivery of CRISPR/Cas9 for therapeutic genome editing

(2019) Xiaojie Xu et al.   JOURNAL OF GENE MEDICINE

Molecular mechanisms of epigenetic inheritance: Possible evolutionary implications

(2019) Peter Sarkies   SEMINARS IN CELL & DEVELOPMENTAL BIOLOGY

dCas9-based Scn1a gene activation restores inhibitory interneuron excitability and attenuates seizures in Dravet syndrome mice

(2019) Gaia Colasante et al.   MOLECULAR THERAPY

The histone mark H3K36me2 recruits DNMT3A and shapes the intergenic DNA methylation landscape

(2019) Daniel N. Weinberg et al.   NATURE

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

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

In Vivo AAV-CRISPR/Cas9-Mediated Gene Editing Ameliorates Atherosclerosis in Familial Hypercholesterolemia

(2019) Huan Zhao et al.   CIRCULATION

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

(2019) Andrew V. Anzalone et al.   NATURE

High levels of AAV vector integration into CRISPR-induced DNA breaks

(2019) Killian S. Hanlon et al.   Nature Communications

In vivo non-invasive monitoring of dystrophin correction in a new Duchenne muscular dystrophy reporter mouse

(2019) Leonela Amoasii et al.   Nature Communications

In vivo epigenetic editing of Sema6a promoter reverses transcallosal dysconnectivity caused by C11orf46/Arl14ep risk gene

(2019) Cyril J. Peter et al.   Nature Communications

Ultrasound-activated particles as CRISPR/Cas9 delivery system for androgenic alopecia therapy

(2019) Jee-Yeon Ryu et al.   BIOMATERIALS

State‐of‐the‐art human adenovirus vectorology for therapeutic approaches

(2019) Jian Gao et al.   FEBS LETTERS

The deletion of mutant SOD1 via CRISPR/Cas9/sgRNA prolongs survival in an amyotrophic lateral sclerosis mouse model

(2019) Weisong Duan et al.   GENE THERAPY

Gene therapy for hemophilia

(2019) Amit C. Nathwani   Hematology-American Society of Hematology Education Program

AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria

(2019) Daelyn Y. Richards et al.   Molecular Therapy-Methods & Clinical Development

Delivering CRISPR: a review of the challenges and approaches

(2018) Christopher A. Lino et al.   DRUG DELIVERY

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

Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity

(2018) Nicole K. Paulk et al.   MOLECULAR THERAPY

CRISPR–Cas9 genome editing induces a p53-mediated DNA damage response

(2018) Emma Haapaniemi et al.   NATURE MEDICINE

CRISPR-LbCpf1 prevents choroidal neovascularization in a mouse model of age-related macular degeneration

(2018) Taeyoung Koo et al.   Nature Communications

CtIP fusion to Cas9 enhances transgene integration by homology-dependent repair

(2018) M. Charpentier et al.   Nature Communications

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

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

VSV-G-Enveloped Vesicles for Traceless Delivery of CRISPR-Cas9

(2018) Claudia Montagna et al.   Molecular Therapy-Nucleic Acids

Virus-like particle vaccines: immunology and formulation for clinical translation

(2018) Braeden Donaldson et al.   Expert Review of Vaccines

Gesicle-Mediated Delivery of CRISPR/Cas9 Ribonucleoprotein Complex for Inactivating the HIV Provirus

(2018) Lee A. Campbell et al.   MOLECULAR THERAPY

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

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

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

Viral vector-based delivery of CRISPR/Cas9 and donor DNA for homology directed repair in an in vitro model for canine hemophilia B

(2018) Jian Gao et al.   Molecular Therapy-Nucleic Acids

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

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

Lipid Nanoparticle Systems for Enabling Gene Therapies

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

CRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10

(2017) Guo-Xiang Ruan et al.   MOLECULAR THERAPY

CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo

(2017) Alex Mas Monteys 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

Cell-Penetrating Peptides: From Basic Research to Clinics

(2017) Giulia Guidotti et al.   TRENDS IN PHARMACOLOGICAL SCIENCES

Genome editing abrogates angiogenesis in vivo

(2017) Xionggao Huang et al.   Nature Communications

Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy

(2017) Niclas E. Bengtsson et al.   Nature Communications

Nrl knockdown by AAV-delivered CRISPR/Cas9 prevents retinal degeneration in mice

(2017) Wenhan Yu et al.   Nature Communications

Integrase-Deficient Lentiviral Vector as an All-in-One Platform for Highly Efficient CRISPR/Cas9-Mediated Gene Editing

(2017) Pavel I. Ortinski et al.   Molecular Therapy-Methods & Clinical Development

Engineering CRISPR–Cpf1 crRNAs and mRNAs to maximize genome editing efficiency

(2017) Bin Li et al.   Nature Biomedical Engineering

Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair

(2017) Kunwoo Lee et al.   Nature Biomedical Engineering

Lentivirus pre-packed with Cas9 protein for safer gene editing

(2016) J G Choi et al.   GENE THERAPY

In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration

(2016) Keiichiro Suzuki et al.   NATURE

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

(2016) Alexis C. Komor et al.   NATURE

A multifunctional AAV–CRISPR–Cas9 and its host response

(2016) Wei Leong Chew et al.   NATURE METHODS

Defining and improving the genome-wide specificities of CRISPR–Cas9 nucleases

(2016) Shengdar Q. Tsai et al.   NATURE REVIEWS GENETICS

CRISPR/Cas9-mediated gene knockout in the mouse brain using in utero electroporation

(2016) Yohei Shinmyo et al.   Scientific Reports

Enrichment of G2/M cell cycle phase in human pluripotent stem cells enhances HDR-mediated gene repair with customizable endonucleases

(2016) Diane Yang et al.   Scientific Reports

Minimizing off-Target Mutagenesis Risks Caused by Programmable Nucleases

(2015) Kentaro Ishida et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Rapid and highly efficient mammalian cell engineering via Cas9 protein transfection

(2015) Xiquan Liang et al.   JOURNAL OF BIOTECHNOLOGY

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Increasing the efficiency of precise genome editing with CRISPR-Cas9 by inhibition of nonhomologous end joining

(2015) Takeshi Maruyama et al.   NATURE BIOTECHNOLOGY

A split-Cas9 architecture for inducible genome editing and transcription modulation

(2015) Bernd Zetsche et al.   NATURE BIOTECHNOLOGY

Functional annotation of native enhancers with a Cas9–histone demethylase fusion

(2015) Nicola A Kearns et al.   NATURE METHODS

Highly efficient Cas9-mediated transcriptional programming

(2015) Alejandro Chavez et al.   NATURE METHODS

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

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

Genome-Scale CRISPR-Mediated Control of Gene Repression and Activation

(2014) Luke A. Gilbert et al.   CELL

Zinc Finger Nucleases Targeting the Human Papillomavirus E7 Oncogene Induce E7 Disruption and a Transformed Phenotype in HPV16/18-Positive Cervical Cancer Cells

(2014) W. Ding et al.   CLINICAL CANCER RESEARCH

Adenovirus: The First Effective In Vivo Gene Delivery Vector

(2014) Ronald G. Crystal   HUMAN GENE THERAPY

TALEN-mediated targeting of HPV oncogenes ameliorates HPV-related cervical malignancy

(2014) Zheng Hu et al.   JOURNAL OF CLINICAL INVESTIGATION

Preclinical research: Make mouse studies work

(2014) Steve Perrin   NATURE

Single nucleotide variations: Biological impact and theoretical interpretation

(2014) Panagiotis Katsonis et al.   PROTEIN SCIENCE

Mechanisms of epigenetic memory

(2014) Agustina D’Urso et al.   TRENDS IN GENETICS

AAV-Mediated Gene Therapy for Research and Therapeutic Purposes

(2014) R. Jude Samulski et al.   Annual Review of Virology

Understanding ultrasound induced sonoporation: Definitions and underlying mechanisms

(2013) I. Lentacker et al.   ADVANCED DRUG DELIVERY REVIEWS

An estimation of the number of cells in the human body

(2013) Eva Bianconi et al.   ANNALS OF HUMAN BIOLOGY

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

(2013) F. Mingozzi et al.   BLOOD

CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes

(2013) Luke A. Gilbert et al.   CELL

High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells

(2013) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer

(2012) G. Buchlis et al.   BLOOD

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

(2012) M A Bartel et al.   GENE THERAPY

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

(2012) M. Jinek et al.   SCIENCE

A 10 Patient Case Report on the Impact of Plasmapheresis Upon Neutralizing Factors Against Adeno-associated Virus (AAV) Types 1, 2, 6, and 8

(2011) Virginie Monteilhet et al.   MOLECULAR THERAPY

High-frequency genome editing using ssDNA oligonucleotides with zinc-finger nucleases

(2011) Fuqiang Chen et al.   NATURE METHODS

AAV-mediated gene targeting methods for human cells

(2011) Iram F Khan et al.   Nature Protocols

Regulation of Homologous Recombination in Eukaryotes

(2010) Wolf-Dietrich Heyer et al.   Annual Review of Genetics

Distinct Epigenomic Landscapes of Pluripotent and Lineage-Committed Human Cells

(2010) R. David Hawkins et al.   Cell Stem Cell

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

Twenty years of cell-penetrating peptides: from molecular mechanisms to therapeutics

(2009) Frederic Heitz et al.   BRITISH JOURNAL OF PHARMACOLOGY

Effect of Genome Size on AAV Vector Packaging

(2009) Zhijian Wu et al.   MOLECULAR THERAPY

Epidemic Keratoconjunctivitis Due to the Novel Hexon-Chimeric-Intermediate 22,37/H8 Human Adenovirus

(2008) K. Aoki et al.   JOURNAL OF CLINICAL MICROBIOLOGY