Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina

Split Cas9, Not Hairs − Advancing the Therapeutic Index of CRISPR Technology

(2018) Carolin Schmelas et al.   Biotechnology Journal

Triple Vectors Expand AAV Transfer Capacity in the Retina

(2018) Andrea Maddalena et al.   MOLECULAR THERAPY

FDA approves hereditary blindness gene therapy

(2018)   NATURE BIOTECHNOLOGY

Seeing the Light after 25 Years of Retinal Gene Therapy

(2018) Ivana Trapani et al.   TRENDS IN MOLECULAR MEDICINE

Split Cas9, Not Hairs − Advancing the Therapeutic Index of CRISPR Technology

(2018) Carolin Schmelas et al.   Biotechnology Journal

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

(2018) Lukas Villiger et al.   NATURE MEDICINE

The Future Looks Brighter After 25 Years of Retinal Gene Therapy

(2017) Alberto Auricchio et al.   HUMAN GENE THERAPY

Split-inteins and their bioapplications

(2015) Yifeng Li   BIOTECHNOLOGY LETTERS

Protein misfolding and the pathogenesis of ABCA4-associated retinal degenerations

(2015) Ning Zhang et al.   HUMAN MOLECULAR GENETICS

Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease

(2015) Ivana Trapani et al.   HUMAN MOLECULAR GENETICS

Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors

(2014) P Colella et al.   GENE THERAPY

Protein Splicing: How Inteins Escape from Precursor Proteins

(2014) Kenneth V. Mills et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Enigmatic Distribution, Evolution, and Function of Inteins

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

Traceless Splicing Enabled by Substrate-Induced Activation of the Nostoc punctiforme Npu DnaE Intein after Mutation of a Catalytic Cysteine to Serine

(2014) Manoj Cheriyan et al.   JOURNAL OF MOLECULAR BIOLOGY

Generation of three-dimensional retinal tissue with functional photoreceptors from human iPSCs

(2014) Xiufeng Zhong et al.   Nature Communications

Dual Adeno-Associated Virus Vectors Result in Efficient In Vitro and In Vivo Expression of an Oversized Gene, MYO7A

(2014) Frank M. Dyka et al.   Human Gene Therapy Methods

Effective delivery of large genes to the retina by dual AAV vectors

(2013) Ivana Trapani et al.   EMBO Molecular Medicine

Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus

(2013) V S Lopes et al.   GENE THERAPY

Disruption of CEP290 microtubule/membrane-binding domains causes retinal degeneration

(2013) Theodore G. Drivas et al.   JOURNAL OF CLINICAL INVESTIGATION

Extein Residues Play an Intimate Role in the Rate-Limiting Step of Protein Trans-Splicing

(2013) Neel H. Shah et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Manipulating L-type calcium channels in cardiomyocytes using split-intein protein transsplicing

(2013) P. Subramanyam et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Inter-chain disulfide bond improved protein trans-splicing increases plasma coagulation activity in C57BL/6 mice following portal vein FVIII gene delivery by dual vectors

(2013) FuXiang Zhu et al.   Science China-Life Sciences

Inteins: nature's gift to protein chemists

(2013) Neel H. Shah et al.   Chemical Science

Self-Formation of Optic Cups and Storable Stratified Neural Retina from Human ESCs

(2012) Tokushige Nakano et al.   Cell Stem Cell

AAV-mediated photoreceptor transduction of the pig cone-enriched retina

(2011) C Mussolino et al.   GENE THERAPY

Protein trans-splicing based dual-vector delivery of the coagulation factor VIII gene

(2010) FuXiang Zhu et al.   Science China-Life Sciences

The naturally splitNpuDnaE intein exhibits an extraordinarily high rate in the proteintrans-splicing reaction

(2009) Joachim Zettler et al.   FEBS LETTERS

Protein Trans-Splicing as a Means for Viral Vector-Mediated In Vivo Gene Therapy

(2008) Juan Li et al.   HUMAN GENE THERAPY