Citation Impact
Citing Papers
An Autophagy-Enhancing Drug Promotes Degradation of Mutant α 1 -Antitrypsin Z and Reduces Hepatic Fibrosis
2010 Science
Prospective identification of tumorigenic breast cancer cells
2003 Standout
MicroRNAs Modulate Hematopoietic Lineage Differentiation
2003 StandoutScience
TFEB Links Autophagy to Lysosomal Biogenesis
2011 StandoutScience
Gene Dose of Apolipoprotein E Type 4 Allele and the Risk of Alzheimer's Disease in Late Onset Families
1993 StandoutScience
A sex-biased imbalance between Tfr, Tph, and atypical B cells determines antibody responses in COVID-19 patients
2023 StandoutNobel
Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency
2017
Hierarchical Mechanisms for Direct Reprogramming of Fibroblasts to Neurons
2013 StandoutNobel
AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes
2015
Genetic determinants of hepatic steatosis in man
2011
Plasmapheresis Eliminates the Negative Impact of AAV Antibodies on Microdystrophin Gene Expression Following Vascular Delivery
2013
Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses
2008
Direct conversion of mouse fibroblasts to hepatocyte-like cells by defined factors
2011 Nature
The ploidy conveyor of mature hepatocytes as a source of genetic variation
2010 Nature
Parvovirus glycan interactions
2014
Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver
2010
Stem cells, cancer, and cancer stem cells
2001 StandoutNature
Analysis of any point mutation in DNA. The amplification refractory mutation system (ARMS)
1989 Standout
Bone marrow cells regenerate infarcted myocardium
2001 StandoutNature
Vascularized and functional human liver from an iPSC-derived organ bud transplant
2013 StandoutNature
Gefitinib or Carboplatin–Paclitaxel in Pulmonary Adenocarcinoma
2009 Standout
Instability and decay of the primary structure of DNA
1993 StandoutNatureNobel
Organoid Models and Applications in Biomedical Research
2015 Standout
State-of-the-art gene-based therapies: the road ahead
2011
Dystrophin Immunity in Duchenne's Muscular Dystrophy
2010
Haematopoietic stem cells do not transdifferentiate into cardiac myocytes in myocardial infarcts
2004 StandoutNature
Cell fusion is the principal source of bone-marrow-derived hepatocytes
2003 Nature
The CpG dinucleotide and human genetic disease
1988
Correction of liver disease by hepatocyte transplantation in a mouse model of progressive familial intrahepatic cholestasis
2000
Mutations in the DNA ligase I gene of an individual with immunodeficiencies and cellular hypersensitivity to DNA-damaging agents
1992 StandoutNobel
Positional cloning of the mouse obese gene and its human homologue
1994 StandoutNature
Intracellular Disposal of Incompletely Folded Human α1-Antitrypsin Involves Release from Calnexin and Post-translational Trimming of Asparagine-linked Oligosaccharides
1997
Robust expansion of human hepatocytes in Fah−/−/Rag2−/−/Il2rg−/− mice
2007
Kinetics of Liver Repopulation after Bone Marrow Transplantation
2002
Induction of functional hepatocyte-like cells from mouse fibroblasts by defined factors
2011 Nature
Hermansky–Pudlak syndrome: a disease of protein trafficking and organelle function
2006
Fah Knockout Animals as Models for Therapeutic Liver Repopulation
2017
Evolution of a designed protein assembly encapsulating its own RNA genome
2017 StandoutNatureNobel
Constitutive Protease-activated Receptor-2-mediated Migration of MDA MB-231 Breast Cancer Cells Requires Both β-Arrestin-1 and -2
2004 StandoutNobel
Gene Therapy With Regulatory T Cells: A Beneficial Alliance
2018
Nonsense Mutations in ADTB3A Cause Complete Deficiency of the β3A Subunit of Adaptor Complex-3 and Severe Hermansky-Pudlak Syndrome Type 2
2002
Crystal structure of the Hhal DNA methyltransferase complexed with S-adenosyl-l-methionine
1993 StandoutNobel
A Serpin Shapes the Extracellular Environment to Prevent Influenza A Virus Maturation
2015 StandoutNobel
Modeling inherited metabolic disorders of the liver using human induced pluripotent stem cells
2010
In vivo genome editing using Staphylococcus aureus Cas9
2015 StandoutNature
CpG-depleted adeno-associated virus vectors evade immune detection
2013
The potential of adeno-associated viral vectors for gene delivery to muscle tissue
2014
In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector
2015
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges
2011
Myelomonocytic cells are sufficient for therapeutic cell fusion in liver
2004
Adeno-associated virus vector as a platform for gene therapy delivery
2019 Standout
Inflammatory Cytokine TNFα Promotes the Long-Term Expansion of Primary Hepatocytes in 3D Culture
2018
Long-Term Expansion of Functional Mouse and Human Hepatocytes as 3D Organoids
2018 StandoutNobel
Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector
2011
Molecular chaperones in cellular protein folding
1996 StandoutNature
Sustained survival of human hepatocytes in mice: A model for in vivo infection with human hepatitis B and hepatitis delta viruses
2000
Surrogate Wnt agonists that phenocopy canonical Wnt and β-catenin signalling
2017 StandoutNatureNobel
The Repopulation Potential of Hepatocyte Populations Differing in Size and Prior Mitotic Expansion
1999
Manufacturing of recombinant adeno-associated viral vectors for clinical trials
2016
Recombinant AAV Viral Vectors Pseudotyped with Viral Capsids from Serotypes 1, 2, and 5 Display Differential Efficiency and Cell Tropism after Delivery to Different Regions of the Central Nervous System
2004
Vascular damage without hypertension in transgenic rats expressing prorenin exclusively in the liver.
1996
Induced pluripotent stem cell–derived hepatocytes have the functional and proliferative capabilities needed for liver regeneration in mice
2010 StandoutNobel
Direct Reprogramming of Human Fibroblasts to Functional and Expandable Hepatocytes
2014
Cerebral organoids model human brain development and microcephaly
2013 StandoutNature
In Vivo Genetic Selection of Renal Proximal Tubules
2005
NF-κB functions as a tumour promoter in inflammation-associated cancer
2004 StandoutNature
Hepatocyte Transplantation for the Treatment of Human Disease
1999
Hepatocyte transplantation in a model of toxin-induced liver disease: variable therapeutic effect during replacement of damaged parenchyma by donor cells
2000
Separation of adeno-associated virus type 2 empty particles from genome containing vectors by anion-exchange column chromatography
2006
Intracellular protein trafficking defects in human disease
1992
Bone marrow cells adopt the phenotype of other cells by spontaneous cell fusion
2002 StandoutNature
Purified hematopoietic stem cells can differentiate into hepatocytes in vivo
2000 Standout
Diagnosis of α1antitrypsin deficiency by enzymatic amplification of human genomic DNA and direct sequencing of polymerase chain reaction products
1988
Unraveling the Complex Story of Immune Responses to AAV Vectors Trial After Trial
2017
In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency
2018
Amelioration of Alpha-1 Antitrypsin Deficiency Diseases with Genome Editing in Transgenic Mice
2018
Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial
2008
Recombinant Adeno-Associated Virus Integration Sites in Murine Liver After Ornithine Transcarbamylase Gene Correction
2013
Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN
2018
Optogenetic Dissection of Entorhinal-Hippocampal Functional Connectivity
2013 StandoutScienceNobel
Slc15a4, AP-3, and Hermansky-Pudlak syndrome proteins are required for Toll-like receptor signaling in plasmacytoid dendritic cells
2010 StandoutNobel
The role of chemokines and chemokine receptors in pulmonary arterial hypertension
2019
Serial transplantation reveals the stem-cell-like regenerative potential of adult mouse hepatocytes.
1997
Mice with mutations of Dock7 have generalized hypopigmentation and white-spotting but show normal neurological function
2009 StandoutNobel
Correction of factor IX deficiency in mice by embryonic stem cells differentiated in vitro
2005 StandoutNobel
A Century of Alzheimer's Disease
2006 StandoutScience
Modeling host interactions with hepatitis B virus using primary and induced pluripotent stem cell-derived hepatocellular systems
2014 StandoutNobel
Modeling hepatitis C virus infection using human induced pluripotent stem cells
2012 StandoutNobel
Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning
2015
β-arrestin- but not G protein-mediated signaling by the “decoy” receptor CXCR7
2009 StandoutNobel
Innate Defense against Influenza A Virus: Activity of Human Neutrophil Defensins and Interactions of Defensins with Surfactant Protein D
2006
Evolution of the genome and the genetic code: selection at the dinucleotide level by methylation and polyribonucleotide cleavage.
1989 StandoutNobel
Infection of Endothelium With E1 − E4 + , but Not E1 − E4 − , Adenovirus Gene Transfer Vectors Enhances Leukocyte Adhesion and Migration by Modulation of ICAM-1, VCAM-1, CD34, and Chemokine Expression
2001
Bipotential Adult Liver Progenitors Are Derived from Chronically Injured Mature Hepatocytes
2014
Tissue Engineering--Current Challenges and Expanding Opportunities
2002 StandoutScience
Intracellular Functions of N-Linked Glycans
2001 StandoutScience
A genetically clamped renin transgene for the induction of hypertension
2002 StandoutNobel
Direct conversion of mouse fibroblasts to self-renewing, tripotent neural precursor cells
2012 StandoutNobel
Pre-existing Anti–Adeno-Associated Virus Antibodies as a Challenge in AAV Gene Therapy
2013
Donor-dependent variations in hepatic differentiation from human-induced pluripotent stem cells
2012 StandoutNobel
Candidate Gene for the Chromosome 1 Familial Alzheimer's Disease Locus
1995 StandoutScience
Liver Regeneration
1997 StandoutScience
Genetic Dissection of Complex Traits
1994 StandoutScience
Directed differentiation of human embryonic stem cells into functional hepatic cells†
2007
The Role of DNA Methylation in Mammalian Epigenetics
2001 StandoutScience
Improved gene expression upon transfer of the adenosine deaminase minigene outside the transcriptional unit of a retroviral vector.
1989
Human Fatty Liver Disease: Old Questions and New Insights
2011 StandoutScience
Synergistic effect of HIF-1α gene therapy and HIF-1-activated bone marrow-derived angiogenic cells in a mouse model of limb ischemia
2009 StandoutNobel
Genetic Linkage Evidence for a Familial Alzheimer's Disease Locus on Chromosome 14
1992 Science
Intranasal Antibody Gene Transfer in Mice and Ferrets Elicits Broad Protection Against Pandemic Influenza
2013
Transduction of Receptor Signals by ß-Arrestins
2005 StandoutScienceNobel
Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape
2017
Works of Mark Brantly being referenced
Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Alpha 1-Antitrypsin (rAAV2-CB-hAAT) Gene Vector to AAT-Deficient Adults
2004
Myeloid-derived Suppressor Cells Are Necessary for Development of Pulmonary Hypertension
2017
Pulmonary Function and High-Resolution CT Findings in Patients With an Inherited Form of Pulmonary Fibrosis, Hermansky-Pudlak Syndrome, Due to Mutations in HPS-1
2000
Hepatocytes corrected by gene therapy are selected in vivo in a murine model of hereditary tyrosinaemia type I
1996
Genetic Defects and Clinical Characteristics of Patients with a Form of Oculocutaneous Albinism (Hermansky–Pudlak Syndrome)
1998
Effect of pirfenidone on the pulmonary fibrosis of Hermansky–Pudlak syndrome
2002
Asthma Features in Severe α1-Antitrypsin Deficiencya
2003
Single nucleotide polymorphism–mediated translational suppression of endoplasmic reticulum mannosidase I modifies the onset of end-stage liver disease in alpha1-antitrypsin deficiency #
2009
Molecular basis of alpha-1-antitrypsin deficiency
1988
Role of human neutrophil peptides in lung inflammation associated with α1-antitrypsin deficiency
2004
The Diagnosis and Management of Alpha-1 Antitrypsin Deficiency in the Adult
2016
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
2009
Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α 1 -Antitrypsin: Interim Results
2011
Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Serotype 2 α 1-Antitrypsin (AAT) Vector in AAT-Deficient Adults
2006
Molecular Basis of the Liver and Lung Disease Associated with the α1-Antitrypsin Deficiency Allele Mmalton
1989
Characterization of the gene and protein of the common alpha 1-antitrypsin normal M2 allele.
1988
Hermansky-Pudlak Syndrome: Radiography and CT of the Chest Compared with Pulmonary Function Tests and Genetic Studies
2002
Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors
1998
Emphysema associated with complete absence of alpha 1- antitrypsin in serum and the homozygous inheritance [corrected] of a stop codon in an alpha 1-antitrypsin-coding exon.
1988
Production of glycosylated physiologically "normal" human alpha 1-antitrypsin by mouse fibroblasts modified by insertion of a human alpha 1-antitrypsin cDNA using a retroviral vector.
1987
Molecular basis of alpha-1-antitrypsin deficiency
1988
Evaluation of "at risk" alpha 1-antitrypsin genotype SZ with synthetic oligonucleotide gene probes.
1986
alpha 1-Antitrypsin nullGranite Falls, a nonexpressing alpha 1-antitrypsin gene associated with a frameshift to stop mutation in a coding exon.
1987
Does the Heterozygous State of Alpha‐1 Antitrypsin Deficiency Have a Role in Chronic Liver Diseases? Interim Results of a Large Case‐Control Study
2006
Identification of a second mutation in the protein-coding sequence of the Z type alpha 1-antitrypsin gene.
1986
Repair of the Secretion Defect in the Z Form of α1-Antitrypsin by Addition of a Second Mutation
1988 Science