Development of Highly Efficient Dual‐AAV Split Adenosine Base Editor for In Vivo Gene Therapy

Engineering adeno-associated virus vectors for gene therapy

(2020) Chengwen Li et al.   NATURE REVIEWS GENETICS

Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses

(2020) Jonathan M. Levy et al.   Nature Biomedical Engineering

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

(2019) Puping Liang et al.   Nature Communications

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

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

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

(2019) Daesik Kim et al.   NATURE BIOTECHNOLOGY

Knockout of Nr2e3 prevents rod photoreceptor differentiation and leads to selective L-/M-cone photoreceptor degeneration in zebrafish

(2019) Shanglun Xie et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Adenine base editing in an adult mouse model of tyrosinaemia

(2019) Chun-Qing Song et al.   Nature Biomedical Engineering

Transcriptome-wide off-target RNA editing induced by CRISPR-guided DNA base editors

(2019) Julian Grünewald et al.   NATURE

In vivo genome editing rescues photoreceptor degeneration via a Cas9/RecA-mediated homology-directed repair pathway

(2019) Yuan Cai et al.   Science Advances

Off-target RNA mutation induced by DNA base editing and its elimination by mutagenesis

(2019) Changyang Zhou et al.   NATURE

CRISPR DNA base editors with reduced RNA off-target and self-editing activities

(2019) Julian Grünewald et al.   NATURE BIOTECHNOLOGY

Engineered Cas9 variant tools expand targeting scope of genome and base editing in rice

(2019) Dongchang Zeng et al.   PLANT BIOTECHNOLOGY JOURNAL

CRISPR-Cas9–mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis

(2019) Dong Hyun Jo et al.   Science Advances

Gene therapy approaches targeting Schwann cells for demyelinating neuropathies

(2019) Irene Sargiannidou et al.   BRAIN RESEARCH

Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy

(2018) Seuk-Min Ryu et al.   NATURE BIOTECHNOLOGY

In vivo base editing of post-mitotic sensory cells

(2018) Wei-Hsi Yeh et al.   Nature Communications

Gene Therapy via Trans -Splicing for LMNA -Related Congenital Muscular Dystrophy

(2018) Feriel Azibani et al.   Molecular Therapy-Nucleic Acids

Evolved Cas9 variants with broad PAM compatibility and high DNA specificity

(2018) Johnny H. Hu et al.   NATURE

Gene therapy for visual loss: Opportunities and concerns

(2018) Jia Hui Lee et al.   PROGRESS IN RETINAL AND EYE RESEARCH

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

(2018) Leonela Amoasii et al.   SCIENCE

Effective and precise adenine base editing in mouse zygotes

(2018) Puping Liang et al.   Protein & Cell

Treatment of a metabolic liver disease by in vivo genome base editing in adult mice

(2018) Lukas Villiger et al.   NATURE MEDICINE

Altered EZH2 splicing and expression is associated with impaired histone H3 lysine 27 tri-Methylation in myelodysplastic syndrome

(2017) Mizuho Shirahata-Adachi et al.   LEUKEMIA RESEARCH

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

(2017) Guo-Xiang Ruan 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

In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni

(2017) Eunji Kim et al.   Nature Communications

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

(2017) Wenhan Yu 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

In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa

(2016) Benjamin Bakondi et al.   MOLECULAR THERAPY

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

(2016) Keiichiro Suzuki et al.   NATURE

A multifunctional AAV–CRISPR–Cas9 and its host response

(2016) Wei Leong Chew et al.   NATURE METHODS

Integration and exchange of split dCas9 domains for transcriptional controls in mammalian cells

(2016) Dacheng Ma et al.   Nature Communications

In vivo Editing of the Human Mutant Rhodopsin Gene by Electroporation of Plasmid-based CRISPR/Cas9 in the Mouse Retina

(2016) Maria Carmela Latella et al.   Molecular Therapy-Nucleic Acids

Cysteine-free non-canonical C-intein for versatile protein C-terminal labeling through trans-splicing

(2015) Xudong Dai et al.   APPLIED MICROBIOLOGY AND BIOTECHNOLOGY

Split-inteins and their bioapplications

(2015) Yifeng Li   BIOTECHNOLOGY LETTERS

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

(2015) Bernd Zetsche et al.   NATURE BIOTECHNOLOGY

Photoactivatable CRISPR-Cas9 for optogenetic genome editing

(2015) Yuta Nihongaki et al.   NATURE BIOTECHNOLOGY

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

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

Trans-spliced Cas9 allows cleavage of HBB and CCR5 genes in human cells using compact expression cassettes

(2015) Eli J. Fine et al.   Scientific Reports

An Evolved Mxe GyrA Intein for Enhanced Production of Fusion Proteins

(2014) Carrie J. Marshall et al.   ACS Chemical Biology

Enigmatic Distribution, Evolution, and Function of Inteins

(2014) Olga Novikova et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial

(2014) Robert E MacLaren et al.   LANCET

Cleaving RNA with Cas9

(2014) Odelia Ghodsizadeh   NATURE METHODS

Nature's recipe for splitting inteins

(2014) A. S. Aranko et al.   PROTEIN ENGINEERING DESIGN & SELECTION

Faster Protein Splicing with theNostoc punctiformeDnaE Intein Using Non-native Extein Residues

(2013) Manoj Cheriyan et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

A survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with Nine natural adeno-associated virus (AAV1-9) and one engineered adeno-associated virus serotype

(2013) Brian L Ellis et al.   Virology Journal

Site-Specific Chemical Modification of Proteins with a Prelabelled Cysteine Tag Using the Artificially Split Mxe GyrA Intein

(2008) Thomas Kurpiers et al.   CHEMBIOCHEM

Dual role of Nr2e3 in photoreceptor development and maintenance

(2008) Andrea L. Webber et al.   EXPERIMENTAL EYE RESEARCH

AAV-mediated gene therapy for retinal disorders: from mouse to man

(2008) P K Buch et al.   GENE THERAPY