In utero gene editing for monogenic lung disease

In vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency

(2018) Chun-Qing Song et al.   HUMAN GENE THERAPY

Expression of mutant Sftpc in murine alveolar epithelia drives spontaneous lung fibrosis

(2018) Shin-Ichi Nureki et al.   JOURNAL OF CLINICAL INVESTIGATION

Erratum: Repair of double-strand breaks induced by CRISPR–Cas9 leads to large deletions and complex rearrangements

(2018) Michael Kosicki et al.   NATURE BIOTECHNOLOGY

Widespread airway distribution and short-term phenotypic correction of cystic fibrosis pigs following aerosol delivery of piggyBac/adenovirus

(2018) Ashley L Cooney et al.   NUCLEIC ACIDS RESEARCH

In utero CRISPR-mediated therapeutic editing of metabolic genes

(2018) Avery C. Rossidis et al.   NATURE MEDICINE

Outcomes of Lung Transplantation for Infants and Children with Genetic Disorders of Surfactant Metabolism

(2017) Whitney B. Eldridge et al.   JOURNAL OF PEDIATRICS

Genome editing reveals a role for OCT4 in human embryogenesis

(2017) Norah M. E. Fogarty et al.   NATURE

Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing

(2017) Hao Yin et al.   NATURE BIOTECHNOLOGY

Interstitial lung disease in newborns

(2017) Lawrence M. Nogee   Seminars in Fetal & Neonatal Medicine

In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni

(2017) Eunji Kim et al.   Nature Communications

Induction of Immune Tolerance to Foreign Protein via Adeno-Associated Viral Vector Gene Transfer in Mid-Gestation Fetal Sheep

(2017) Marcus G. Davey et al.   PLoS One

CRISPR-Cas9 mediated one-step disabling of pancreatogenesis in pigs

(2017) Jun Wu et al.   Scientific Reports

Epidemiology of Cystic Fibrosis

(2016) Kimberly A. Spoonhower et al.   CLINICS IN CHEST MEDICINE

HDAC3-Dependent Epigenetic Pathway Controls Lung Alveolar Epithelial Cell Remodeling and Spreading via miR-17-92 and TGF-β Signaling Regulation

(2016) Yi Wang et al.   DEVELOPMENTAL CELL

The Mucus Barrier to Inhaled Gene Therapy

(2016) Gregg A Duncan et al.   MOLECULAR THERAPY

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

(2016) Yang Yang et al.   NATURE BIOTECHNOLOGY

Defining and improving the genome-wide specificities of CRISPR–Cas9 nucleases

(2016) Shengdar Q. Tsai et al.   NATURE REVIEWS GENETICS

A non-BRICHOS SFTPC mutant (SP-CI73T) linked to interstitial lung disease promotes a late block in macroautophagy disrupting cellular proteostasis and mitophagy

(2015) Arie Hawkins et al.   AMERICAN JOURNAL OF PHYSIOLOGY-LUNG CELLULAR AND MOLECULAR PHYSIOLOGY

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

(2015) M. Tabebordbar et al.   SCIENCE

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

(2015) C. E. Nelson et al.   SCIENCE

Immunological Ignorance Allows Long-Term Gene Expression After Perinatal Recombinant Adeno-Associated Virus-Mediated Gene Transfer to Murine Airways

(2014) Marianne S. Carlon et al.   HUMAN GENE THERAPY

Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA

(2014) C. Long et al.   SCIENCE

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

In Utero Lung Gene Transfer Using Adeno-Associated Viral and Lentiviral Vectors in Mice

(2014) Luc Joyeux et al.   Human Gene Therapy Methods

Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9

(2013) Yuxuan Wu et al.   Cell Stem Cell

Cas9 as a versatile tool for engineering biology

(2013) Prashant Mali et al.   NATURE METHODS

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Adenovirus receptors: implications for targeting of viral vectors

(2012) Niklas Arnberg   TRENDS IN PHARMACOLOGICAL SCIENCES

Adenoviral Vector Immunity: Its Implications and Circumvention Strategies

(2011) Yadvinder S. Ahi et al.   CURRENT GENE THERAPY

A Nonaggregating Surfactant Protein C Mutant Is Misdirected to Early Endosomes and Disrupts Phospholipid Recycling

(2011) Michael F. Beers et al.   TRAFFIC

Alveolar Surfactant Homeostasis and the Pathogenesis of Pulmonary Disease

(2010) Jeffrey A. Whitsett et al.   Annual Review of Medicine

Progress and Prospects: prospects of repeated pulmonary administration of viral vectors

(2009) P L Sinn et al.   GENE THERAPY

The developmental stage determines the distribution and duration of gene expression after early intra-amniotic gene transfer using lentiviral vectors

(2009) M Endo et al.   GENE THERAPY

Clinical course and prognosis of never-smokers with severe alpha-1-antitrypsin deficiency (PiZZ)

(2008) H A Tanash et al.   THORAX