Agammaglobulinemia: causative mutations and their implications for novel therapies

Genome Engineering at the Dawn of the Golden Age

(2013) David J. Segal et al.   Annual Review of Genomics and Human Genetics

Development of gene therapy for blood disorders: an update

(2013) A. W. Nienhuis   BLOOD

A Phenotypic Approach for IUIS PID Classification and Diagnosis: Guidelines for Clinicians at the Bedside

(2013) Ahmed Aziz Bousfiha et al.   JOURNAL OF CLINICAL IMMUNOLOGY

Gene therapy clinical trials worldwide to 2012 - an update

(2013) Samantha L. Ginn et al.   JOURNAL OF GENE MEDICINE

Splicing therapy for neuromuscular disease

(2013) Andrew G.L. Douglas et al.   MOLECULAR AND CELLULAR NEUROSCIENCE

Genomic Editing of the HIV-1 Coreceptor CCR5 in Adult Hematopoietic Stem and Progenitor Cells Using Zinc Finger Nucleases

(2013) Lijing Li et al.   MOLECULAR THERAPY

Fyn Kinase Is Required for Optimal Humoral Responses

(2013) Natalia S. Chaimowitz et al.   PLoS One

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Evaluating risks of insertional mutagenesis by DNA transposons in gene therapy

(2013) Perry B. Hackett et al.   Translational Research

Targeting RNA splicing for disease therapy

(2013) Mallory A. Havens et al.   Wiley Interdisciplinary Reviews-RNA

Induced pluripotent stem cells in clinical hematology

(2012) Athanasia D. Panopoulos et al.   CURRENT OPINION IN HEMATOLOGY

Recent developments in transposon-mediated gene therapy

(2012) Mario Di Matteo et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs

(2012) B L Ellis et al.   GENE THERAPY

Precision genome engineering with programmable DNA-nicking enzymes

(2012) E. Kim et al.   GENOME RESEARCH

An exon-specific U1 small nuclear RNA (snRNA) strategy to correct splicing defects

(2012) Eugenio Fernandez Alanis et al.   HUMAN MOLECULAR GENETICS

Agammaglobulinemia and absent B lineage cells in a patient lacking the p85α subunit of PI3K

(2012) Mary Ellen Conley et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Alpharetroviral Self-inactivating Vectors: Long-term Transgene Expression in Murine Hematopoietic Cells and Low Genotoxicity

(2012) Julia D Suerth et al.   MOLECULAR THERAPY

The promise of induced pluripotent stem cells in research and therapy

(2012) Daisy A. Robinton et al.   NATURE

Cold Urticaria, Immunodeficiency, and Autoimmunity Related toPLCG2Deletions

(2012) Michael J. Ombrello et al.   NEW ENGLAND JOURNAL OF MEDICINE

Akt and mTOR in B Cell Activation and Differentiation

(2012) Jose J. Limon et al.   Frontiers in Immunology

Agammaglobulinemia associated with BCR- B cells and enhanced expression of CD19

(2011) A. K. Dobbs et al.   BLOOD

IL-7R expression and IL-7 signaling confer a distinct phenotype on developing human B-lineage cells

(2011) S. E. Nodland et al.   BLOOD

Epigenetic Memory and Preferential Lineage-Specific Differentiation in Induced Pluripotent Stem Cells Derived from Human Pancreatic Islet Beta Cells

(2011) Ori Bar-Nur et al.   Cell Stem Cell

Key aspects for successful immunoglobulin therapy of primary immunodeficiencies

(2011) C. Cunningham-Rundles   CLINICAL AND EXPERIMENTAL IMMUNOLOGY

Blk haploinsufficiency impairs the development, but enhances the functional responses, of MZ B cells

(2011) Elizabeth M Samuelson et al.   IMMUNOLOGY AND CELL BIOLOGY

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

(2011) Sebahattin Cirak et al.   LANCET

Regulation of BCR signaling

(2011) Tomohiro Kurosaki   MOLECULAR IMMUNOLOGY

Immunogenicity of induced pluripotent stem cells

(2011) Tongbiao Zhao et al.   NATURE

In vivo genome editing restores haemostasis in a mouse model of haemophilia

(2011) Hojun Li et al.   NATURE

Genetic engineering of human pluripotent cells using TALE nucleases

(2011) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

Site-specific integration and tailoring of cassette design for sustainable gene transfer

(2011) Angelo Lombardo et al.   NATURE METHODS

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

(2011) Nathalie M. Goemans et al.   NEW ENGLAND JOURNAL OF MEDICINE

Design principles for bifunctional targeted oligonucleotide enhancers of splicing

(2011) N. Owen et al.   NUCLEIC ACIDS RESEARCH

Compensatory signals associated with the activation of human GC 5′ splice sites

(2011) Jana Kralovicova et al.   NUCLEIC ACIDS RESEARCH

Targeted Genome Editing Across Species Using ZFNs and TALENs

(2011) Andrew J. Wood et al.   SCIENCE

B cell-specific lentiviral gene therapy leads to sustained B-cell functional recovery in a murine model of X-linked agammaglobulinemia

(2010) H. M. Kerns et al.   BLOOD

Identification and Classification of Chromosomal Aberrations in Human Induced Pluripotent Stem Cells

(2010) Yoav Mayshar et al.   Cell Stem Cell

Correction of B-cell development in Btk-deficient mice using lentiviral vectors with codon-optimized human BTK

(2010) Y Y Ng et al.   LEUKEMIA

Development of B-lineage Predominant Lentiviral Vectors for Use in Genetic Therapies for B Cell Disorders

(2010) Blythe D Sather et al.   MOLECULAR THERAPY

20 years of gene therapy for SCID

(2010) Alain Fischer et al.   NATURE IMMUNOLOGY

Primary B Cell Immunodeficiencies: Comparisons and Contrasts

(2009) Mary Ellen Conley et al.   Annual Review of Immunology

Rescue of coagulation factor VII function by the U1+5A snRNA

(2009) M. Pinotti et al.   BLOOD

A critical role of TAK1 in B-cell receptor–mediated nuclear factor κB activation

(2009) James Schuman et al.   BLOOD

Foxo3-/- mice demonstrate reduced numbers of pre-B and recirculating B cells but normal splenic B cell sub-population distribution

(2009) R. M. Hinman et al.   INTERNATIONAL IMMUNOLOGY

Gross Deletions Involving IGHM, BTK, or Artemis: A Model for Genomic Lesions Mediated by Transposable Elements

(2008) Menno C. van Zelm et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Development of an enhanced B-specific lentiviral vector expressing BTK: a tool for gene therapy of XLA

(2008) T Moreau et al.   GENE THERAPY

Erk Kinases Link Pre-B Cell Receptor Signaling to Transcriptional Events Required for Early B Cell Expansion

(2008) Tomoharu Yasuda et al.   IMMUNITY

Distinct functions for the transcription factor Foxo1 at various stages of B cell differentiation

(2008) Hart S Dengler et al.   NATURE IMMUNOLOGY

Characterization of Bruton's tyrosine kinase mutations in Mexican patients with X-linked agammaglobulinemia

(2007) G. Lopez-Herrera et al.   MOLECULAR IMMUNOLOGY