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

Modified Poly(lactic-co-glycolic Acid) Nanoparticles for Enhanced Cellular Uptake and Gene Editing in the Lung

(2014) Rachel J. Fields et al.   Advanced Healthcare Materials

Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients

(2013) Gerald Schwank et al.   Cell Stem Cell

Minimal "Self" Peptides That Inhibit Phagocytic Clearance and Enhance Delivery of Nanoparticles

(2013) P. L. Rodriguez et al.   SCIENCE

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

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

Surface modified poly(β amino ester)-containing nanoparticles for plasmid DNA delivery

(2012) Rachel J. Fields et al.   JOURNAL OF CONTROLLED RELEASE

Advances in Cell and Gene-based Therapies for Cystic Fibrosis Lung Disease

(2012) Mayumi Oakland et al.   MOLECULAR THERAPY

Fast gapped-read alignment with Bowtie 2

(2012) Ben Langmead et al.   NATURE METHODS

A microRNA network regulates expression and biosynthesis of wild-type and  F508 mutant cystic fibrosis transmembrane conductance regulator

(2012) S. Ramachandran et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Nanoparticle-based therapy in an in vivo microRNA-155 (miR-155)-dependent mouse model of lymphoma

(2012) I. A. Babar et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Nanomaterials for Drug Delivery

(2012) J. A. Hubbell et al.   SCIENCE

Preclinical Development and Clinical Translation of a PSMA-Targeted Docetaxel Nanoparticle with a Differentiated Pharmacological Profile

(2012) J. Hrkach et al.   Science Translational Medicine

Generation and Genetic Engineering of Human Induced Pluripotent Stem Cells Using Designed Zinc Finger Nucleases

(2012) Sivaprakash Ramalingam et al.   STEM CELLS AND DEVELOPMENT

Enhanced siRNA delivery into cells by exploiting the synergy between targeting ligands and cell-penetrating peptides

(2011) Christopher J. Cheng et al.   BIOMATERIALS

Btrim: A fast, lightweight adapter and quality trimming program for next-generation sequencing technologies

(2011) Yong Kong   GENOMICS

Mouse models of cystic fibrosis: Phenotypic analysis and research applications

(2011) Martina Wilke et al.   Journal of Cystic Fibrosis

Calcium-Modulated Chloride Pathways Contribute to Chloride Flux in Murine Cystic Fibrosis-Affected Macrophages

(2011) Ambika Shenoy et al.   PEDIATRIC RESEARCH

The  F508 Mutation Causes CFTR Misprocessing and Cystic Fibrosis-Like Disease in Pigs

(2011) L. S. Ostedgaard et al.   Science Translational Medicine

Role of epithelial HCO3− transport in mucin secretion: lessons from cystic fibrosis

(2010) Paul M. Quinton   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY

An Update on Cystic Fibrosis Screening

(2010) Katherine R. Goetzinger et al.   CLINICS IN LABORATORY MEDICINE

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

(2010) Nicole A McNeer et al.   MOLECULAR THERAPY

Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo

(2010) Nathalia Holt et al.   NATURE BIOTECHNOLOGY

Adenovirus 5–Fiber 35 Chimeric Vector Mediates Efficient Apical Correction of the Cystic Fibrosis Transmembrane Conductance Regulator Defect in Cystic Fibrosis Primary Airway Epithelia

(2009) Ophélia Granio et al.   HUMAN GENE THERAPY

ELDA: Extreme limiting dilution analysis for comparing depleted and enriched populations in stem cell and other assays

(2009) Yifang Hu et al.   JOURNAL OF IMMUNOLOGICAL METHODS

Partial Correction of Cystic Fibrosis Defects with PLGA Nanoparticles Encapsulating Curcumin

(2009) Malgorzata S. Cartiera et al.   MOLECULAR PHARMACEUTICS

Reduced histone deacetylase 7 activity restores function to misfolded CFTR in cystic fibrosis

(2009) Darren M Hutt et al.   Nature Chemical Biology

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

(2009) Alessandro Aiuti et al.   NEW ENGLAND JOURNAL OF MEDICINE

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

(2009) N. Cartier et al.   SCIENCE

Gene transfer to the lung: Lessons learned from more than 2 decades of CF gene therapy

(2008) Uta Griesenbach et al.   ADVANCED DRUG DELIVERY REVIEWS

Macrophages Directly Contribute to the Exaggerated Inflammatory Response in Cystic Fibrosis Transmembrane Conductance Regulator−/−Mice

(2008) Emanuela M. Bruscia et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Cystic fibrosis: impaired bicarbonate secretion and mucoviscidosis

(2008) Paul M Quinton   LANCET

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