In utero nanoparticle delivery for site-specific genome editing

Peptide Nucleic Acids as a Tool for Site-Specific Gene Editing

(2018) Adele Ricciardi et al.   MOLECULES

Nanoparticle targeting to the endothelium during normothermic machine perfusion of human kidneys

(2017) Gregory T. Tietjen et al.   Science Translational Medicine

In utero stem cell transplantation and gene therapy: Recent progress and the potential for clinical application

(2016) Lauren E. McClain et al.   BEST PRACTICE & RESEARCH CLINICAL OBSTETRICS & GYNAECOLOGY

Improved i.p. drug delivery with bioadhesive nanoparticles

(2016) Yang Deng et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery

(2016) Raman Bahal et al.   Nature Communications

CRISPR-Cas9 for in vivo Gene Therapy: Promise and Hurdles

(2016) Wei-Jing Dai et al.   Molecular Therapy-Nucleic Acids

In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical application

(2016) Graça Almeida-Porada et al.   Molecular Therapy-Methods & Clinical Development

Challenges in CRISPR/CAS9 Delivery: Potential Roles of Nonviral Vectors

(2015) Ling Li et al.   HUMAN GENE THERAPY

Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium

(2015) Nicole Ali McNeer et al.   Nature Communications

Off-target Effects in CRISPR/Cas9-mediated Genome Engineering

(2015) Xiao-Hui Zhang et al.   Molecular Therapy-Nucleic Acids

Development of the Fetal Bone Marrow Niche and Regulation of HSC Quiescence and Homing Ability by Emerging Osteolineage Cells

(2014) Süleyman Coşkun et al.   Cell Reports

PEAR: a fast and accurate Illumina Paired-End reAd mergeR

(2013) J. Zhang et al.   BIOINFORMATICS

Quantitative analysis of murine terminal erythroid differentiation in vivo: novel method to study normal and disordered erythropoiesis

(2013) J. Liu et al.   BLOOD

Site-specific Genome Editing in PBMCs With PLGA Nanoparticle-delivered PNAs Confers HIV-1 Resistance in Humanized Mice

(2013) Erica B Schleifman et al.   Molecular Therapy-Nucleic Acids

Systemic delivery of triplex-forming PNA and donor DNA by nanoparticles mediates site-specific genome editing of human hematopoietic cells in vivo

(2012) N A McNeer et al.   GENE THERAPY

Non-invasive prenatal measurement of the fetal genome

(2012) H. Christina Fan et al.   NATURE

CD71 is Selectively and Ubiquitously Expressed at High Levels in Erythroid Precursors of All Maturation Stages

(2011) Henry Y. Dong et al.   AMERICAN JOURNAL OF SURGICAL PATHOLOGY

Sequence-Unrestricted, Watson-Crick Recognition of Double Helical B-DNA by (R)-MiniPEG-γPNAs

(2011) Raman Bahal et al.   CHEMBIOCHEM

Cell cycle regulation in hematopoietic stem cells

(2011) Eric M. Pietras et al.   JOURNAL OF CELL BIOLOGY

Synthesis and Characterization of Conformationally Preorganized, (R)-Diethylene Glycol-Containing γ-Peptide Nucleic Acids with Superior Hybridization Properties and Water Solubility

(2011) Bichismita Sahu et al.   JOURNAL OF ORGANIC CHEMISTRY

Fast and accurate long-read alignment with Burrows–Wheeler transform

(2010) Heng Li et al.   BIOINFORMATICS

Pure erythroid leukemia: a reassessment of the entity using the 2008 World Health Organization classification

(2010) Wei Liu et al.   MODERN PATHOLOGY

Nanoparticles Deliver Triplex-forming PNAs for Site-specific Genomic Recombination in CD34+ Human Hematopoietic Progenitors

(2010) Nicole A McNeer et al.   MOLECULAR THERAPY

Resolving the distinct stages in erythroid differentiation based on dynamic changes in membrane protein expression during erythropoiesis

(2009) K. Chen et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Stem cell and genetic therapies for the fetus

(2009) Jessica L. Roybal et al.   Seminars in Fetal & Neonatal Medicine

Global epidemiology of haemoglobin disorders and derived service indicators

(2008) Bernadette Modell   BULLETIN OF THE WORLD HEALTH ORGANIZATION

Hematopoiesis: An Evolving Paradigm for Stem Cell Biology

(2008) Stuart H. Orkin et al.   CELL

A Simple Cytosine to G-Clamp Nucleobase Substitution Enables Chiral γ-PNAs to Invade Mixed-Sequence Double-Helical B-form DNA

(2008) Venugopal Chenna et al.   CHEMBIOCHEM

Repair of DNA lesions associated with triplex-forming oligonucleotides

(2008) Joanna Y. Chin et al.   MOLECULAR CARCINOGENESIS

Of lineage and legacy: the development of mammalian hematopoietic stem cells

(2008) Elaine Dzierzak et al.   NATURE IMMUNOLOGY

Correction of a splice-site mutation in the beta-globin gene stimulated by triplex-forming peptide nucleic acids

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

In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of  -thalassemia

(2008) A. Miccio et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA