Published in Mol Ther on October 23, 2012
Nephropathic cystinosis: an international consensus document. Nephrol Dial Transplant (2014) 1.14
Brief reports: Lysosomal cross-correction by hematopoietic stem cell-derived macrophages via tunneling nanotubes. Stem Cells (2015) 0.89
A mouse model suggests two mechanisms for thyroid alterations in infantile cystinosis: decreased thyroglobulin synthesis due to endoplasmic reticulum stress/unfolded protein response and impaired lysosomal processing. Endocrinology (2015) 0.86
Cystinosis: a review. Orphanet J Rare Dis (2016) 0.85
Management dilemmas in pediatric nephrology: Cystinosis. Pediatr Nephrol (2015) 0.84
Cell-based therapies for experimental chronic kidney disease: a systematic review and meta-analysis. Dis Model Mech (2015) 0.84
Lysosome dysfunction in the pathogenesis of kidney diseases. Pediatr Nephrol (2013) 0.78
Treatment of Inherited Eye Defects by Systemic Hematopoietic Stem Cell Transplantation. Invest Ophthalmol Vis Sci (2015) 0.78
Clinical utility of chitotriosidase enzyme activity in nephropathic cystinosis. Orphanet J Rare Dis (2014) 0.78
Is genetic rescue of cystinosis an achievable treatment goal? Nephrol Dial Transplant (2013) 0.76
Hematopoietic Stem Cells Transplantation Can Normalize Thyroid Function in a Cystinosis Mouse Model. Endocrinology (2016) 0.76
The renal Fanconi syndrome in cystinosis: pathogenic insights and therapeutic perspectives. Nat Rev Nephrol (2016) 0.75
Cystinosin, the small GTPase Rab11, and the Rab7 effector RILP regulate intracellular trafficking of the chaperone-mediated autophagy receptor LAMP2A. J Biol Chem (2017) 0.75
Improving the prognosis of nephropathic cystinosis. Int J Nephrol Renovasc Dis (2014) 0.75
Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications. Mol Ther (2017) 0.75
That eagle covering me: transitioning and connected autonomy for emerging adults with cystinosis. Pediatr Nephrol (2014) 0.75
Gyrfalcons Falco rusticolus adjust CTNS expression to food abundance: a possible contribution to cysteine homeostasis. Oecologia (2017) 0.75
Exosome-mediated transfer of mRNAs and microRNAs is a novel mechanism of genetic exchange between cells. Nat Cell Biol (2007) 37.10
In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science (1996) 28.95
A third-generation lentivirus vector with a conditional packaging system. J Virol (1998) 16.07
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest (2008) 9.42
Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science (2009) 8.80
Gene therapy -- promises, problems and prospects. Nature (1997) 6.02
Cystinosis. N Engl J Med (2002) 4.51
Exosomes/microvesicles as a mechanism of cell-to-cell communication. Kidney Int (2010) 4.13
The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy. J Clin Invest (2009) 3.34
A novel gene encoding an integral membrane protein is mutated in nephropathic cystinosis. Nat Genet (1998) 3.24
Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science (1999) 3.10
Structurally distinct membrane nanotubes between human macrophages support long-distance vesicular traffic or surfing of bacteria. J Immunol (2006) 2.65
Physiological promoters reduce the genotoxic risk of integrating gene vectors. Mol Ther (2008) 2.56
Quantitative analysis reveals expansion of human hematopoietic repopulating cells after short-term ex vivo culture. J Exp Med (1997) 2.03
Long-term follow-up of well-treated nephropathic cystinosis patients. J Pediatr (2004) 2.02
Exosomes: Fit to deliver small RNA. Commun Integr Biol (2010) 1.93
Gene therapy progress and prospects: development of improved lentiviral and retroviral vectors--design, biosafety, and production. Gene Ther (2005) 1.93
Alteration of marrow cell gene expression, protein production, and engraftment into lung by lung-derived microvesicles: a novel mechanism for phenotype modulation. Stem Cells (2007) 1.92
Cystinosin, the protein defective in cystinosis, is a H(+)-driven lysosomal cystine transporter. EMBO J (2001) 1.83
Stable transduction of quiescent CD34(+)CD38(-) human hematopoietic cells by HIV-1-based lentiviral vectors. Proc Natl Acad Sci U S A (1999) 1.80
Nanotubes, exosomes, and nucleic acid-binding peptides provide novel mechanisms of intercellular communication in eukaryotic cells: implications in health and disease. J Cell Biol (2008) 1.77
Assay of human stem cells by repopulation of NOD/SCID mice. Stem Cells (1997) 1.74
Kidney preservation by bone marrow cell transplantation in hereditary nephropathy. Kidney Int (2011) 1.72
Bone marrow transplantation for globoid cell leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy, and Hurler syndrome. Curr Opin Hematol (1999) 1.59
Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells. J Clin Invest (2004) 1.59
Paracrine/endocrine mechanism of stem cells on kidney repair: role of microvesicle-mediated transfer of genetic information. Curr Opin Nephrol Hypertens (2010) 1.42
The targeting of cystinosin to the lysosomal membrane requires a tyrosine-based signal and a novel sorting motif. J Biol Chem (2001) 1.33
Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model. Blood (2010) 1.33
Successful treatment of the murine model of cystinosis using bone marrow cell transplantation. Blood (2009) 1.28
Intralysosomal cystine accumulation in mice lacking cystinosin, the protein defective in cystinosis. Mol Cell Biol (2002) 1.25
Long-term outcome of nephropathic cystinosis: a 20-year single-center experience. Pediatr Nephrol (2010) 1.17
Advances in lentiviral vector design for gene-modification of hematopoietic stem cells. Curr Opin Biotechnol (2002) 1.13
Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector. Hum Gene Ther (2005) 1.12
Acute graft-versus-host disease: differing risk with differing graft sources and conditioning intensity. Best Pract Res Clin Haematol (2008) 1.12
Hematopoietic stem cell transplantation: an overview of infection risks and epidemiology. Infect Dis Clin North Am (2010) 1.04
Unaltered repopulation properties of mouse hematopoietic stem cells transduced with lentiviral vectors. Blood (2008) 1.02
Calcineurin inhibitor-free GVHD prophylaxis with sirolimus, mycophenolate mofetil and ATG in Allo-SCT for leukemia patients with high relapse risk: an observational cohort study. Bone Marrow Transplant (2008) 1.01
Retroviral insertion site analysis in dominant haematopoietic clones. Methods Mol Biol (2009) 0.98
A comparison of immune reconstitution and graft-versus-host disease following myeloablative conditioning versus reduced toxicity conditioning and umbilical cord blood transplantation in paediatric recipients. Br J Haematol (2011) 0.94
Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype. Blood (2010) 0.92
Challenges and advances in infection control of hematopoietic stem cell transplant recipients. Infect Disord Drug Targets (2011) 0.89
Cysteamine therapy: a treatment for cystinosis, not a cure. Kidney Int (2012) 0.88
Metabolic correction in microglia derived from Sandhoff disease model mice. J Neurochem (2005) 0.87
Gene transfer may be preventive but not curative for a lysosomal transport disorder. Mol Ther (2008) 0.80
Retrovirus vector-mediated correction and cross-correction of lysosomal alpha-mannosidase deficiency in human and feline fibroblasts. Hum Gene Ther (1999) 0.79
Managing the toxicity of hematopoietic stem cell transplant. J Support Oncol (2004) 0.79
Long-term expression of the human glucocerebrosidase gene in vivo after transplantation of bone-marrow-derived cells transformed with a lentivirus vector. J Gene Med (2005) 0.78
Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nat Biotechnol (2010) 6.55
Redirecting specificity of T-cell populations for CD19 using the Sleeping Beauty system. Cancer Res (2008) 2.57
Genetic therapies against HIV. Nat Biotechnol (2007) 2.50
Kidney transplant rejection and tissue injury by gene profiling of biopsies and peripheral blood lymphocytes. Am J Transplant (2004) 2.35
De novo kidney transplantation without use of calcineurin inhibitors preserves renal structure and function at two years. Am J Transplant (2004) 2.17
Strategies for aggregating gene expression data: the collapseRows R function. BMC Bioinformatics (2011) 1.96
Transient gene expression by nonintegrating lentiviral vectors. Mol Ther (2006) 1.89
Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans. Blood (2012) 1.85
Transplantation outcomes for severe combined immunodeficiency, 2000-2009. N Engl J Med (2014) 1.81
Dynamic tracking of human hematopoietic stem cell engraftment using in vivo bioluminescence imaging. Blood (2003) 1.79
Kidney preservation by bone marrow cell transplantation in hereditary nephropathy. Kidney Int (2011) 1.72
Marrow-derived cells as vehicles for delivery of gene therapy to pulmonary epithelium. Am J Respir Cell Mol Biol (2002) 1.70
Newborn screening for severe combined immunodeficiency and T-cell lymphopenia in California: results of the first 2 years. J Allergy Clin Immunol (2013) 1.66
Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates. Nat Med (2003) 1.65
Antitumor activity from antigen-specific CD8 T cells generated in vivo from genetically engineered human hematopoietic stem cells. Proc Natl Acad Sci U S A (2011) 1.61
Systemic immunoregulatory and proteogenomic effects of tacrolimus to sirolimus conversion in liver transplant recipients. Hepatology (2012) 1.56
Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012. Hum Gene Ther (2013) 1.54
Expansion of multipotent and lymphoid-committed human progenitors through intracellular dimerization of Mpl. Blood (2008) 1.51
A modified γ-retrovirus vector for X-linked severe combined immunodeficiency. N Engl J Med (2014) 1.42
Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID). Clin Immunol (2005) 1.40
The natural history of children with severe combined immunodeficiency: baseline features of the first fifty patients of the primary immune deficiency treatment consortium prospective study 6901. J Clin Immunol (2013) 1.40
Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency. J Clin Invest (2017) 1.39
Prostate cancer originating in basal cells progresses to adenocarcinoma propagated by luminal-like cells. Proc Natl Acad Sci U S A (2013) 1.37
Finding the active genes in deep RNA-seq gene expression studies. BMC Genomics (2013) 1.35
Factors influencing the titer and infectivity of lentiviral vectors. Hum Gene Ther (2004) 1.32
American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells. Mol Ther (2003) 1.29
Successful treatment of the murine model of cystinosis using bone marrow cell transplantation. Blood (2009) 1.28
Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(+) cells. Mol Ther (2005) 1.28
Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: current status and critical needs. J Allergy Clin Immunol (2008) 1.25
Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector. Mol Ther (2005) 1.25
Quantitative bias in Illumina TruSeq and a novel post amplification barcoding strategy for multiplexed DNA and small RNA deep sequencing. PLoS One (2011) 1.24
Gene therapy fulfilling its promise. N Engl J Med (2009) 1.24
Identification of receptors for pig endogenous retrovirus. Proc Natl Acad Sci U S A (2003) 1.23
Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management. J Allergy Clin Immunol (2009) 1.23
β-globin gene transfer to human bone marrow for sickle cell disease. J Clin Invest (2013) 1.22
MicroRNA regulation of molecular networks mapped by global microRNA, mRNA, and protein expression in activated T lymphocytes. J Immunol (2011) 1.18
The Moloney murine leukemia virus repressor binding site represses expression in murine and human hematopoietic stem cells. J Virol (2003) 1.17
Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow. Mol Genet Metab (2003) 1.15
Generation and characterization of transgene-free human induced pluripotent stem cells and conversion to putative clinical-grade status. Stem Cell Res Ther (2013) 1.14
In vivo biosafety model to assess the risk of adverse events from retroviral and lentiviral vectors. Mol Ther (2008) 1.14
Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction. Blood (2012) 1.14
Intrapulmonary and intramyocardial gene transfer in rhesus monkeys (Macaca mulatta): safety and efficiency of HIV-1-derived lentiviral vectors for fetal gene delivery. Mol Ther (2005) 1.14
Advances in lentiviral vector design for gene-modification of hematopoietic stem cells. Curr Opin Biotechnol (2002) 1.13
Stable gene transfer to human CD34(+) hematopoietic cells using the Sleeping Beauty transposon. Exp Hematol (2006) 1.13
Molecular mechanisms of chronic kidney transplant rejection via large-scale proteogenomic analysis of tissue biopsies. J Am Soc Nephrol (2010) 1.13
Method for improved Illumina sequencing library preparation using NuGEN Ovation RNA-Seq System. Biotechniques (2011) 1.10
The role of angiopoietins in the development of endothelial cells from cord blood CD34+ progenitors. Blood (2004) 1.09
Integrase-defective lentiviral vectors as a delivery platform for targeted modification of adenosine deaminase locus. Mol Ther (2013) 1.08
Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB. Mol Genet Metab (2004) 1.08
Stable transgene expression in primitive human CD34+ hematopoietic stem/progenitor cells, using the Sleeping Beauty transposon system. Hum Gene Ther (2009) 1.08
From skin biopsy to neurons through a pluripotent intermediate under Good Manufacturing Practice protocols. Stem Cells Transl Med (2011) 1.07
Integrated self-inactivating lentiviral vectors produce full-length genomic transcripts competent for encapsidation and integration. J Virol (2004) 1.06
Highly efficient large-scale lentiviral vector concentration by tandem tangential flow filtration. J Virol Methods (2011) 1.06
Biomarkers for early and late stage chronic allograft nephropathy by proteogenomic profiling of peripheral blood. PLoS One (2009) 1.03
Deconvoluting post-transplant immunity: cell subset-specific mapping reveals pathways for activation and expansion of memory T, monocytes and B cells. PLoS One (2010) 1.03
Transduction of green fluorescent protein increased oxidative stress and enhanced sensitivity to cytotoxic drugs in neuroblastoma cell lines. Mol Cancer Ther (2003) 1.03
PhenoChipping of psychotic disorders: a novel approach for deconstructing and quantitating psychiatric phenotypes. Am J Med Genet B Neuropsychiatr Genet (2006) 1.02
Morphological analysis and lentiviral transduction of fetal monkey bone marrow-derived mesenchymal stem cells. Mol Ther (2004) 1.01
Adoptive transfer of MART-1 T-cell receptor transgenic lymphocytes and dendritic cell vaccination in patients with metastatic melanoma. Clin Cancer Res (2014) 1.01
Application of microdroplet PCR for large-scale targeted bisulfite sequencing. Genome Res (2011) 1.01
Manipulation of OCT4 levels in human embryonic stem cells results in induction of differential cell types. Exp Biol Med (Maywood) (2007) 1.00
Modification of hematopoietic stem/progenitor cells with CD19-specific chimeric antigen receptors as a novel approach for cancer immunotherapy. Hum Gene Ther (2013) 1.00
Allelic exclusion and peripheral reconstitution by TCR transgenic T cells arising from transduced human hematopoietic stem/progenitor cells. Mol Ther (2013) 1.00
Effective suicide gene therapy for leukemia in a model of insertional oncogenesis in mice. Mol Ther (2007) 0.99
Progressive declines in neurocognitive function among survivors of hematopoietic stem cell transplantation for pediatric hematologic malignancies. J Pediatr Hematol Oncol (2008) 0.99
Porcine endogenous retrovirus infects but does not replicate in nonhuman primate primary cells and cell lines. J Virol (2002) 0.98
Lentivirus vectors incorporating the immunoglobulin heavy chain enhancer and matrix attachment regions provide position-independent expression in B lymphocytes. J Virol (2003) 0.98
Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy. Mol Ther (2012) 0.98
The infectivity and host range of the ecotropic porcine endogenous retrovirus, PERV-C, is modulated by residues in the C-terminal region of its surface envelope protein. Virology (2005) 0.98
Lentiviral vectors ready for prime-time. Nat Biotechnol (2007) 0.97
Genome-wide analysis of immune activation in human T and B cells reveals distinct classes of alternatively spliced genes. PLoS One (2009) 0.97
Effects of busulfan dose escalation on engraftment of infant rhesus monkey hematopoietic stem cells after gene marking by a lentiviral vector. Exp Hematol (2006) 0.97
Ectopic B-cell clusters that infiltrate transplanted human kidneys are clonal. Proc Natl Acad Sci U S A (2011) 0.96
Long-term in vivo monitoring of mouse and human hematopoietic stem cell engraftment with a human positron emission tomography reporter gene. Proc Natl Acad Sci U S A (2013) 0.96
Efficient characterization of retro-, lenti-, and foamyvector-transduced cell populations by high-accuracy insertion site sequencing. Ann N Y Acad Sci (2003) 0.96
Why are some amyloidoses systemic? Does hepatic "chaperoning at a distance" prevent cardiac deposition in a transgenic model of human senile systemic (transthyretin) amyloidosis? FASEB J (2012) 0.96
The class I HLA repertoire of pancreatic islets comprises the nonclassical class Ib antigen HLA-G. Diabetes (2006) 0.96
Hematopoietic stem cells for cancer immunotherapy. Immunol Rev (2014) 0.95
Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report. Blood (2006) 0.95
Lentiviral vector transduction of a dominant-negative Rev gene into human CD34+ hematopoietic progenitor cells potently inhibits human immunodeficiency virus-1 replication. Mol Ther (2007) 0.94
Genomic biomarkers correlate with HLA-identical renal transplant tolerance. J Am Soc Nephrol (2013) 0.94
Kidney repair and stem cells: a complex and controversial process. Pediatr Nephrol (2011) 0.94
An association of candidate gene haplotypes and bleeding severity in von Willebrand disease (VWD) type 1 pedigrees. Blood (2004) 0.93
Pseudotyping of porcine endogenous retrovirus by xenotropic murine leukemia virus in a pig islet xenotransplantation model. Am J Transplant (2005) 0.93
Fetal gene transfer using lentiviral vectors and the potential for germ cell transduction in rhesus monkeys (Macaca mulatta). Hum Gene Ther (2005) 0.93
Specific and stable gene transfer to human embryonic stem cells using pseudotyped lentiviral vectors. Stem Cells Dev (2006) 0.92
Applying genomics to organ transplantation medicine in both discovery and validation of biomarkers. Int Immunopharmacol (2007) 0.92
Antibodies reactive to non-HLA antigens in transplant glomerulopathy. J Am Soc Nephrol (2011) 0.91
A tale of two SCIDs. Sci Transl Med (2011) 0.91
Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency. Blood (2011) 0.90
CD4+CD25- T cells transduced to express MHC class I-restricted epitope-specific TCR synthesize Th1 cytokines and exhibit MHC class I-restricted cytolytic effector function in a human melanoma model. J Immunol (2008) 0.90
Gene therapy of RAG-2-/- mice: sustained correction of the immunodeficiency. Blood (2002) 0.90
Expression patterns and function of chromatin protein HMGB2 during mesenchymal stem cell differentiation. J Biol Chem (2011) 0.90
Activation of counter-regulatory mechanisms in a rat renal acute rejection model. BMC Genomics (2008) 0.89
Brief reports: Lysosomal cross-correction by hematopoietic stem cell-derived macrophages via tunneling nanotubes. Stem Cells (2015) 0.89