Related references
Note: Only part of the references are listed.Restricted spacer tolerance of a zinc finger nuclease with a six amino acid linker
Yuka Shimizu et al.
BIOORGANIC & MEDICINAL CHEMISTRY LETTERS (2009)
Gene therapy of inherited skin adhesion disorders: a critical overview
M. De Luca et al.
BRITISH JOURNAL OF DERMATOLOGY (2009)
Engineered Lentiviral Vector Targeting Astrocytes In Vivo
Angelique Colin et al.
GLIA (2009)
The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy
Eugenio Montini et al.
JOURNAL OF CLINICAL INVESTIGATION (2009)
Mutational Derivatives of PhiC31 Integrase With Increased Efficiency and Specificity
Annahita Keravala et al.
MOLECULAR THERAPY (2009)
A yeast-endonuclease-generated DNA break induces antigenic switching in Trypanosoma brucei
Catharine E. Boothroyd et al.
NATURE (2009)
Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates
Lajos Mates et al.
NATURE GENETICS (2009)
Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells
Ronald T. Mitsuyasu et al.
NATURE MEDICINE (2009)
The emerging role of nuclear architecture in DNA repair and genome maintenance
Tom Misteli et al.
NATURE REVIEWS MOLECULAR CELL BIOLOGY (2009)
Optimization of in vivo activity of a bifunctional homing endonuclease and maturase reverses evolutionary degradation
Ryo Takeuchi et al.
NUCLEIC ACIDS RESEARCH (2009)
Generation of single-chain LAGLIDADG homing endonucleases from native homodimeric precursor proteins
Hui Li et al.
NUCLEIC ACIDS RESEARCH (2009)
Generation of a nicking enzyme that stimulates site-specific gene conversion from the I-AniI LAGLIDADG homing endonuclease
Audrey McConnell Smith et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2009)
Chromosomal translocations induced at specified loci in human stem cells
Erika Brunet et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2009)
Directed evolution of homing endonuclease I-SceI with altered sequence specificity
Zhilei Chen et al.
PROTEIN ENGINEERING DESIGN & SELECTION (2009)
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
Steven J. Howe et al.
JOURNAL OF CLINICAL INVESTIGATION (2008)
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
Salima Hacein-Bey-Abina et al.
JOURNAL OF CLINICAL INVESTIGATION (2008)
Rapid Open-Source engineering of customized zinc-finger nucleases for highly efficient gene modification
Morgan L. Maeder et al.
MOLECULAR CELL (2008)
Molecular basis of xeroderma pigmentosum group C DNA recognition by engineered meganucleases
Pilar Redondo et al.
NATURE (2008)
Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases
Elena E. Perez et al.
NATURE BIOTECHNOLOGY (2008)
Safety and efficacy of gene transfer for Leber's congenital amaurosis
Albert M. Maguire et al.
NEW ENGLAND JOURNAL OF MEDICINE (2008)
Effect of gene therapy on visual function in Leber's congenital amaurosis
James W. B. Bainbridge et al.
NEW ENGLAND JOURNAL OF MEDICINE (2008)
Sequence-specific DNA cleavage mediated by bipyridine polyamide conjugates
Philippe Simon et al.
NUCLEIC ACIDS RESEARCH (2008)
Computer design of obligate heterodimer meganucleases allows efficient cutting of custom DNA sequences
Emmanuel Fajardo-Sanchez et al.
NUCLEIC ACIDS RESEARCH (2008)
Development of a single-chain, quasi-dimeric zinc-finger nuclease for the selective degradation of mutated human mitochondrial DNA
Michal Minczuk et al.
NUCLEIC ACIDS RESEARCH (2008)
Group II Intron-Based Gene Targeting Reactions in Eukaryotes
Marta Mastroianni et al.
PLOS ONE (2008)
Crystal structure of I-Dmol in complex with its target DNA provides new insights into meganuclease engineering
Maria Jose Marciada et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2008)
被撤回的出版物: Triplex-forming oligonucleotide-orthophenanthroline conjugates for efficient targeted genome modification (Retracted article. See vol. 107, pg. 5261, 2010)
Fabio Cannata et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2008)
Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin
Jacob Hanna et al.
SCIENCE (2007)
Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state
Brian D. Brown et al.
NATURE BIOTECHNOLOGY (2007)
Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery
Angelo Lombardo et al.
NATURE BIOTECHNOLOGY (2007)
Phage phiC31 integrase-mediated genomic integration and long-term gene expression in the lung after nonviral gene delivery
Manish Kumar Aneja et al.
JOURNAL OF GENE MEDICINE (2007)
Altered target site specificity variants of the I-PpoI His-Cys box homing endonuclease
Jennifer L. Eklund et al.
NUCLEIC ACIDS RESEARCH (2007)
Engineered I-Crel derivatives cleaving sequences from the human XPC gene can induce highly efficient gene correction in mammalian cells
Sylvain Arnould et al.
JOURNAL OF MOLECULAR BIOLOGY (2007)
Structure-based redesign of the dimerization interface reduces the toxicity of zinc-finger nucleases
Michal Szczepek et al.
NATURE BIOTECHNOLOGY (2007)
An improved zinc-finger nuclease architecture for highly specific genome editing
Jeffrey C. Miller et al.
NATURE BIOTECHNOLOGY (2007)
HIV-1 proviral DNA excision using an evolved recombinase
Indrani Sarkar et al.
SCIENCE (2007)
Targeted Sleeping Beauty transposition in human cells
Zoltan Ivics et al.
MOLECULAR THERAPY (2007)
The C-terminal loop of the homing endonuclease I-CreI is essential for site recognition, DNA binding and cleavage
Jesus Prieto et al.
NUCLEIC ACIDS RESEARCH (2007)
Meganucleases and DNA double-strand break-induced recombination:: Perspectives for gene therapy
Frederic Paques et al.
CURRENT GENE THERAPY (2007)
The genetic engineering of hematopoietic stem cells: The rise of lentiviral vectors, the conundrum of the LTR, and the promise of lineage-restricted vectors
Alex H. Chang et al.
MOLECULAR THERAPY (2007)
False positive results in chimeraplasty for von Willebrand Disease
Simon F. De Meyer et al.
THROMBOSIS RESEARCH (2007)
A combinatorial approach to create artificial homing endonucleases cleaving chosen sequences
Julianne Smith et al.
NUCLEIC ACIDS RESEARCH (2006)
The φC31 integrase system for gene therapy
Michele P. Calos
CURRENT GENE THERAPY (2006)
Homing endonuclease I-CreI derivatives with novel DNA target specificities
Laura E. Rosen et al.
NUCLEIC ACIDS RESEARCH (2006)
Transposons for gene therapy!
Zoltan Ivics et al.
CURRENT GENE THERAPY (2006)
Correction of sickle cell disease by homologous recombination in embryonic stem cells
Li-Chen Wu et al.
BLOOD (2006)
Targeted gene repair: The ups and downs of a promising gene therapy approach
David de Semir et al.
CURRENT GENE THERAPY (2006)
Computational redesign of endonuclease DNA binding and cleavage specificity
Justin Ashworth et al.
NATURE (2006)
Recent advances in globin gene transfer for the treatment of beta-thalassemia and sickle cell anemia
Michel Sadelain
CURRENT OPINION IN HEMATOLOGY (2006)
Integration specificity of phage φC31 integrase in the human genome
TW Chalberg et al.
JOURNAL OF MOLECULAR BIOLOGY (2006)
Directed evolution and substrate specificity profile of homing endonuclease I-scel
JB Doyon et al.
JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2006)
In vitro functional correction of the mutation responsible for murine severe combined immune deficiency by small fragment homologous replacement
H Zayed et al.
HUMAN GENE THERAPY (2006)
Correction of the sickle cell mutation in embryonic stem cells
JC Chang et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2006)
Engineering of large numbers of highly specific homing endonucleases that induce recombination on novel DNA targets
S Arnould et al.
JOURNAL OF MOLECULAR BIOLOGY (2006)
Exploring the cellular activity of camptothecin-triple-helix-forming oligonucleotide conjugates
PB Arimondo et al.
MOLECULAR AND CELLULAR BIOLOGY (2006)
Silencing and variegation of gammaretrovirus and lentivirus vectors
J Ellis
HUMAN GENE THERAPY (2005)
Factors affecting double-strand break-induced homologous recombination in mammalian cells
C Perez et al.
BIOTECHNIQUES (2005)
Highly efficient endogenous human gene correction using designed zinc-finger nucleases
FD Urnov et al.
NATURE (2005)
Homing endonuclease structure and function
BL Stoddard
QUARTERLY REVIEWS OF BIOPHYSICS (2005)
Zinc finger nucleases: custom-designed molecular scissors for genome engineering of plant and mammalian cells
S Durai et al.
NUCLEIC ACIDS RESEARCH (2005)
Developing a programmed restriction endonuclease for highly specific DNA cleavage
K Eisenschmidt et al.
NUCLEIC ACIDS RESEARCH (2005)
In vivo selection of engineered homing endonucleases using double-strand break induced homologous recombination
P Chames et al.
NUCLEIC ACIDS RESEARCH (2005)
A highly sensitive selection method for directed evolution of homing endonucleases
ZL Chen et al.
NUCLEIC ACIDS RESEARCH (2005)
Sleeping Beauty transposition:: Biology and applications for molecular therapy
Z Izsvák et al.
MOLECULAR THERAPY (2004)
Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector
HB Gaspar et al.
LANCET (2004)
Metal-dependent DNA cleavage mechanism of the I-CreI LAGLIDADG homing endonuclease
B Chevalier et al.
BIOCHEMISTRY (2004)
Efficient lentiviral vector-mediated control of HIV-1 replication in CD4 lymphocytes from diverse HIV+ infected patients grouped according to CD4 count and viral load
LM Humeau et al.
MOLECULAR THERAPY (2004)
Assessing the plasticity of DNA target site recognition of the Pl-Scel homing endonuclease using a bacterial two-hybrid selection system
FS Gimble et al.
JOURNAL OF MOLECULAR BIOLOGY (2003)
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
S Hacein-Bey-Abina et al.
SCIENCE (2003)
Unsuccessful chimeraplast strategy for the correction of a mutation causing Gaucher disease
A Diaz-Font et al.
BLOOD CELLS MOLECULES AND DISEASES (2003)
A novel engineered meganuclease induces homologous recombination in yeast and mammalian cells
JC Epinat et al.
NUCLEIC ACIDS RESEARCH (2003)
Recognition of the DNA minor groove by pyrrole-imidazole polyamides
PB Dervan et al.
CURRENT OPINION IN STRUCTURAL BIOLOGY (2003)
Flexible DNA target site recognition by divergent homing endonuclease isoschizomers I-CreI and I-MsoI
B Chevalier et al.
JOURNAL OF MOLECULAR BIOLOGY (2003)
Drug discovery with engineered zinc-finger proteins
AC Jamieson et al.
NATURE REVIEWS DRUG DISCOVERY (2003)
Stepwise manipulation of DNA specificity in Flp recombinase: Progressively adapting Flp to individual and combinatorial mutations in its target site
Y Voziyanov et al.
JOURNAL OF MOLECULAR BIOLOGY (2003)
Site-specific genomic integration produces therapeutic Factor IX levels in mice
EC Olivares et al.
NATURE BIOTECHNOLOGY (2002)
Common physical properties of DNA affecting target site selection of Sleeping Beauty and other Tc1/mariner transposable elements
TJ Vigdal et al.
JOURNAL OF MOLECULAR BIOLOGY (2002)
I-SceI meganuclease mediates highly efficient transgenesis in fish
V Thermes et al.
MECHANISMS OF DEVELOPMENT (2002)
Design, activity, and structure of a highly specific artificial endonuclease
BS Chevalier et al.
MOLECULAR CELL (2002)
Mutations altering the cleavage specificity of a homing endonuclease
LM Seligman et al.
NUCLEIC ACIDS RESEARCH (2002)
Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning
A Aiuti et al.
SCIENCE (2002)
Targeted gene correction in the mdX mouse using short DNA fragments: towards application with bone marrow-derived cells for autologous remodeling of dystrophic muscle
RM Kapsa et al.
GENE THERAPY (2002)
Directed evolution of the site specificity of Cre recombinase
SW Santoro et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2002)
Group II introns as controllable gene targeting vectors for genetic manipulation of bacteria
M Karberg et al.
NATURE BIOTECHNOLOGY (2001)
Alteration of Cre recombinase site specificity by substrate-linked protein evolution
F Buchholz et al.
NATURE BIOTECHNOLOGY (2001)
Homing endonucleases: structural and functional insight into the catalysts of intron/intein mobility
BS Chevalier et al.
NUCLEIC ACIDS RESEARCH (2001)
Gene correction of the apolipoprotein (apo) E2 phenotype to wild-type apoE3 by in situ chimeraplasty
AD Tagalakis et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2001)
In vivo and in vitro correction of the mdx dystrophin gene nonsense mutation by short-fragment homologous replacement
R Kapsa et al.
HUMAN GENE THERAPY (2001)
Multiple homing pathways used by yeast mitochondrial group II introns
R Eskes et al.
MOLECULAR AND CELLULAR BIOLOGY (2000)
Group II introns designed to insert into therapeutically relevant DNA target sites in human cells
HT Guo et al.
SCIENCE (2000)
Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin
C May et al.
NATURE (2000)
Frequent chromosomal translocations induced by DNA double-strand breaks
C Richardson et al.
NATURE (2000)
Rescue of dystrophin expression in mdx mouse muscle by RNA/DNA oligonucleotides
TA Rando et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2000)
Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
M Cavazzana-Calvo et al.
SCIENCE (2000)
Requirements for double-strand cleavage by chimeric restriction enzymes with zinc finger DNA-recognition domains
J Smith et al.
NUCLEIC ACIDS RESEARCH (2000)