Editing out fiveSerpina1paralogs to create a mouse model of genetic emphysema

Canu: scalable and accurate long-read assembly via adaptivek-mer weighting and repeat separation

(2017) Sergey Koren et al.   GENOME RESEARCH

5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency

(2017) Christian Mueller et al.   MOLECULAR THERAPY

Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency

(2017) Florie Borel et al.   MOLECULAR THERAPY

Imaging of pulmonary emphysema: A pictorial review

(2016) Masashi Takahashi   International Journal of Chronic Obstructive Pulmonary Disease

PacBio-LITS: a large-insert targeted sequencing method for characterization of human disease-associated chromosomal structural variations

(2015) Min Wang et al.   BMC GENOMICS

HTSeq--a Python framework to work with high-throughput sequencing data

(2014) S. Anders et al.   BIOINFORMATICS

Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases

(2014) S. Bae et al.   BIOINFORMATICS

Generating genetically modified mice using CRISPR/Cas-mediated genome engineering

(2014) Hui Yang et al.   Nature Protocols

CRISPRseek: A Bioconductor Package to Identify Target-Specific Guide RNAs for CRISPR-Cas9 Genome-Editing Systems

(2014) Lihua J. Zhu et al.   PLoS One

COSMID: A Web-based Tool for Identifying and Validating CRISPR/Cas Off-target Sites

(2014) Thomas J Cradick et al.   Molecular Therapy-Nucleic Acids

The Electrophoretic α1-Globulin Pattern of Serum in α1-Antitrypsin Deficiency

(2013) Carl-Bertil Laurell et al.   COPD-Journal of Chronic Obstructive Pulmonary Disease

Human Treg responses allow sustained recombinant adeno-associated virus–mediated transgene expression

(2013) Christian Mueller et al.   JOURNAL OF CLINICAL INVESTIGATION

TopHat2: accurate alignment of transcriptomes in the presence of insertions, deletions and gene fusions

(2013) Daehwan Kim et al.   GENOME BIOLOGY

Evaluation of Respiratory System Mechanics in Mice using the Forced Oscillation Technique

(2013) Toby K. McGovern et al.   Jove-Journal of Visualized Experiments

Global and regional mortality from 235 causes of death for 20 age groups in 1990 and 2010: a systematic analysis for the Global Burden of Disease Study 2010

(2012) Rafael Lozano et al.   LANCET

Sustained miRNA-mediated Knockdown of Mutant AAT With Simultaneous Augmentation of Wild-type AAT Has Minimal Effect on Global Liver miRNA Profiles

(2012) Christian Mueller et al.   MOLECULAR THERAPY

clusterProfiler: an R Package for Comparing Biological Themes Among Gene Clusters

(2012) Guangchuang Yu et al.   OMICS-A JOURNAL OF INTEGRATIVE BIOLOGY

Deletion of serpina1a, a murine α1-antitrypsin ortholog, results in embryonic lethality

(2011) Dongmei Wang et al.   EXPERIMENTAL LUNG RESEARCH

Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α1-Antitrypsin: Interim Results

(2011) Terence R. Flotte et al.   HUMAN GENE THERAPY

Age-Dependent Decline in Mouse Lung Regeneration with Loss of Lung Fibroblast Clonogenicity and Increased Myofibroblastic Differentiation

(2011) Julia A. Paxson et al.   PLoS One