Targeted approaches for gene therapy and the emergence of engineered meganucleases

Restricted spacer tolerance of a zinc finger nuclease with a six amino acid linker

(2009) Yuka Shimizu et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Gene therapy of inherited skin adhesion disorders: a critical overview

(2009) M. De Luca et al.   BRITISH JOURNAL OF DERMATOLOGY

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

(2009) Eugenio Montini et al.   JOURNAL OF CLINICAL INVESTIGATION

A yeast-endonuclease-generated DNA break induces antigenic switching in Trypanosoma brucei

(2009) Catharine E. Boothroyd et al.   NATURE

Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates

(2009) Lajos Mátés et al.   NATURE GENETICS

Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells

(2009) Ronald T Mitsuyasu et al.   NATURE MEDICINE

The emerging role of nuclear architecture in DNA repair and genome maintenance

(2009) Tom Misteli et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Generation of single-chain LAGLIDADG homing endonucleases from native homodimeric precursor proteins

(2009) Hui Li et al.   NUCLEIC ACIDS RESEARCH

Generation of a nicking enzyme that stimulates site-specific gene conversion from the I-AniI LAGLIDADG homing endonuclease

(2009) A. McConnell Smith et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Chromosomal translocations induced at specified loci in human stem cells

(2009) E. Brunet et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Directed evolution of homing endonuclease I-SceI with altered sequence specificity

(2009) Z. Chen et al.   PROTEIN ENGINEERING DESIGN & SELECTION

Engineered lentiviral vector targeting astrocytesIn vivo

(2008) Angélique Colin et al.   GLIA

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

(2008) Steven J. Howe et al.   JOURNAL OF CLINICAL INVESTIGATION

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION

Rapid “Open-Source” Engineering of Customized Zinc-Finger Nucleases for Highly Efficient Gene Modification

(2008) Morgan L. Maeder et al.   MOLECULAR CELL

Mutational Derivatives of PhiC31 Integrase With Increased Efficiency and Specificity

(2008) Annahita Keravala et al.   MOLECULAR THERAPY

Molecular basis of xeroderma pigmentosum group C DNA recognition by engineered meganucleases

(2008) Pilar Redondo et al.   NATURE

Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases

(2008) Elena E Perez et al.   NATURE BIOTECHNOLOGY

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

(2008) Albert M. Maguire et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

(2008) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE

Sequence-specific DNA cleavage mediated by bipyridine polyamide conjugates

(2008) Philippe Simon et al.   NUCLEIC ACIDS RESEARCH

Computer design of obligate heterodimer meganucleases allows efficient cutting of custom DNA sequences

(2008) Emmanuel Fajardo-Sanchez et al.   NUCLEIC ACIDS RESEARCH

Development of a single-chain, quasi-dimeric zinc-finger nuclease for the selective degradation of mutated human mitochondrial DNA

(2008) Michal Minczuk et al.   NUCLEIC ACIDS RESEARCH

Optimization of in vivo activity of a bifunctional homing endonuclease and maturase reverses evolutionary degradation

(2008) Ryo Takeuchi et al.   NUCLEIC ACIDS RESEARCH

Group II Intron-Based Gene Targeting Reactions in Eukaryotes

(2008) Marta Mastroianni et al.   PLoS One

Crystal structure of I-DmoI in complex with its target DNA provides new insights into meganuclease engineering

(2008) M. J. Marcaida et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Triplex-forming oligonucleotide-orthophenanthroline conjugates for efficient targeted genome modification

(2008) F. Cannata et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA