| scholarly article | Q13442814 |
| P50 | author | Shawn M. Burgess | Q38154986 |
| Gaurav K. Varshney | Q52674137 | ||
| Matthew C LaFave | Q56911384 | ||
| Nuria Carrillo-Carrasco | Q73881061 | ||
| Randy Chandler | Q79150394 | ||
| P2093 | author name string | Charles P Venditti | |
| Niraj S Trivedi | |||
| Abdel G Elkahloun | |||
| Julien S Senac | |||
| Victoria Hoffmann | |||
| Weiwei Wu | |||
| P2860 | cites work | Ultrafast and memory-efficient alignment of short DNA sequences to the human genome | Q21183894 |
| Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy | Q24535690 | ||
| Safety and efficacy of gene transfer for Leber's congenital amaurosis | Q24634724 | ||
| The human genome browser at UCSC | Q24672361 | ||
| MLV integration site selection is driven by strong enhancers and active promoters | Q37701268 | ||
| Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges | Q37866086 | ||
| Gene therapy clinical trials worldwide to 2012 - an update. | Q38077166 | ||
| Biosafety of recombinant adeno-associated virus vectors. | Q38160261 | ||
| Endocytosis of adeno-associated virus type 5 leads to accumulation of virus particles in the Golgi compartment | Q39748126 | ||
| AAV serotype 2 vectors preferentially integrate into active genes in mice | Q40643545 | ||
| The fetal mouse is a sensitive genotoxicity model that exposes lentiviral-associated mutagenesis resulting in liver oncogenesis | Q41317435 | ||
| Adeno-associated virus capsid proteins may play a role in transcription and second-strand synthesis of recombinant genomes | Q41861397 | ||
| Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union | Q42516930 | ||
| Vector-related tumorigenesis not found in ornithine transcarbamylase-deficient mice | Q44236123 | ||
| Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors. | Q44974380 | ||
| Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver. | Q45225518 | ||
| No tumour-initiating risk associated with scAAV transduction in newborn rat liver. | Q45790615 | ||
| A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency | Q45857796 | ||
| Quest for safety at AAValon | Q45868739 | ||
| AAV vector integration sites in mouse hepatocellular carcinoma. | Q45869780 | ||
| No evidence for tumorigenesis of AAV vectors in a large-scale study in mice. | Q45885410 | ||
| Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice | Q45885733 | ||
| AAV vectors and tumorigenicity | Q46266970 | ||
| Age-related incidence of spontaneous tumors in SPF C57BL/6 and BDF1 mice | Q67857063 | ||
| Spontaneous lesions in aging FVB/N mice | Q71971445 | ||
| AAV vectors, insertional mutagenesis, and cancer | Q81326066 | ||
| Abnormal hepatocellular mitochondria in methylmalonic acidemia | Q88087645 | ||
| Bub1 mediates cell death in response to chromosome missegregation and acts to suppress spontaneous tumorigenesis | Q24682464 | ||
| BLAT—The BLAST-Like Alignment Tool | Q24682492 | ||
| DLK1-DIO3 genomic imprinted microRNA cluster at 14q32.2 defines a stemlike subtype of hepatocellular carcinoma associated with poor survival | Q28242740 | ||
| Pre-clinical efficacy and dosing of an AAV8 vector expressing human methylmalonyl-CoA mutase in a murine model of methylmalonic acidemia (MMA) | Q28276617 | ||
| Effect of gene therapy on visual function in Leber's congenital amaurosis | Q28277981 | ||
| Ensembl 2013 | Q28280494 | ||
| Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response | Q29619095 | ||
| BamTools: a C++ API and toolkit for analyzing and managing BAM files | Q30426432 | ||
| Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correction | Q30496514 | ||
| Sialic acid deposition impairs the utility of AAV9, but not peptide-modified AAVs for brain gene therapy in a mouse model of lysosomal storage disease | Q30519560 | ||
| Metabolic phenotype of methylmalonic acidemia in mice and humans: the role of skeletal muscle | Q33302709 | ||
| Long-term rescue of a lethal murine model of methylmalonic acidemia using adeno-associated viral gene therapy | Q33730476 | ||
| Ensembl BioMarts: a hub for data retrieval across taxonomic space | Q33969847 | ||
| Identification of a new imprinted gene, Rian, on mouse chromosome 12 by fluorescent differential display screening | Q34085531 | ||
| Transcriptome comparison between fetal and adult mouse livers: implications for circadian clock mechanisms | Q34170754 | ||
| rAAV-mediated tumorigenesis: still unresolved after an AAV assault | Q34310293 | ||
| Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial | Q34311144 | ||
| Identification of rtl1, a retrotransposon-derived imprinted gene, as a novel driver of hepatocarcinogenesis | Q34672049 | ||
| Assessing the potential for AAV vector genotoxicity in a murine model | Q34754355 | ||
| Self-complementary AAV vectors; advances and applications | Q34804660 | ||
| Promoterless gene targeting without nucleases ameliorates haemophilia B in mice. | Q34979802 | ||
| Adeno-associated virus type 2 wild-type and vector-mediated genomic integration profiles of human diploid fibroblasts analyzed by third-generation PacBio DNA sequencing | Q35207938 | ||
| Adenovirus-associated virus vector-mediated gene transfer in hemophilia B | Q35691659 | ||
| The AAV vector toolkit: poised at the clinical crossroads | Q35876224 | ||
| Gene therapy in a murine model of methylmalonic acidemia using rAAV9-mediated gene delivery | Q36055431 | ||
| Induction of hepatocellular carcinoma by in vivo gene targeting | Q36094151 | ||
| Patterns of scAAV vector insertion associated with oncogenic events in a mouse model for genotoxicity | Q36401085 | ||
| Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors | Q36509889 | ||
| Lentiviral vector-based insertional mutagenesis identifies genes associated with liver cancer | Q36660711 | ||
| A large-scale zebrafish gene knockout resource for the genome-wide study of gene function | Q36732481 | ||
| Recombinant adeno-associated virus integration sites in murine liver after ornithine transcarbamylase gene correction | Q36848708 | ||
| A modified sleeping beauty transposon system that can be used to model a wide variety of human cancers in mice | Q36980093 | ||
| Mitochondrial dysfunction in mut methylmalonic acidemia | Q37139811 | ||
| Site-specific integration by adeno-associated virus | Q37246710 | ||
| P4510 | describes a project that uses | ImageJ | Q1659584 |
| P433 | issue | 2 | |
| P407 | language of work or name | English | Q1860 |
| P921 | main subject | gene therapy | Q213901 |
| genotoxicity | Q1009245 | ||
| vector-borne disease | Q2083837 | ||
| P304 | page(s) | 870-880 | |
| P577 | publication date | 2015-01-20 | |
| P1433 | published in | Journal of Clinical Investigation | Q3186904 |
| P1476 | title | Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy | |
| P478 | volume | 125 |
| Q91826241 | A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I |
| Q60912963 | A Self-Deleting AAV-CRISPR System for Genome Editing |
| Q102075541 | A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells |
| Q28468539 | A universal system to select gene-modified hepatocytes in vivo |
| Q90732125 | AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice |
| Q40457152 | AAV Infection: Protection from Cancer. |
| Q38789554 | Adeno-Associated Virus Gene Therapy for Liver Disease |
| Q39546902 | Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma? |
| Q42553527 | Adeno-associated Vector Toxicity-To Be or Not to Be? |
| Q36713767 | Adeno-associated viral vectors for the treatment of hemophilia. |
| Q42584196 | Adeno-associated virus finds its disease |
| Q64040883 | Adeno-associated virus vector as a platform for gene therapy delivery |
| Q57178720 | Advances and innovations in haemophilia treatment |
| Q57170592 | Advances in gene therapy for hemophilia: basis, current status, and future perspectives |
| Q93051143 | Blood-Brain Barrier and Delivery of Protein and Gene Therapeutics to Brain |
| Q39294371 | CRISPR/Cas9: at the cutting edge of hepatology. |
| Q64377006 | Conditional disruption of hepatic carbamoyl phosphate synthetase 1 in mice results in hyperammonemia without orotic aciduria and can be corrected by liver-directed gene therapy |
| Q30856034 | Curative ex vivo liver-directed gene therapy in a pig model of hereditary tyrosinemia type 1. |
| Q92017672 | Curing hemophilia A by NHEJ-mediated ectopic F8 insertion in the mouse |
| Q104289829 | Current Clinical Applications of in vivo Gene Therapy with AAVs |
| Q38827606 | Current and future prospects for hemophilia gene therapy |
| Q99724691 | Delivery Approaches for Therapeutic Genome Editing and Challenges |
| Q92535035 | Developing Modern Pain Therapies |
| Q40798280 | Development of an AAV9 coding for a 3XFLAG-TALEfrat#8-VP64 able to increase in vivo the human frataxin in YG8R mice |
| Q47554736 | Emerging Issues in AAV-Mediated In Vivo Gene Therapy |
| Q89777435 | Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors |
| Q40986812 | Establishment of two quantitative nested qPCR assays targeting the human EPO transgene. |
| Q60912810 | FGF21 underlies a hormetic response to metabolic stress in methylmalonic acidemia |
| Q36117412 | GeIST: a pipeline for mapping integrated DNA elements |
| Q99579530 | Gene Therapy for Hemophilia: Facts and Quandaries in the 21st Century |
| Q45874307 | Gene Therapy for Hemophilia: Progress to Date. |
| Q28595868 | Gene Therapy for Metabolic Diseases |
| Q38719347 | Gene therapy decreases seizures in a model of Incontinentia pigmenti. |
| Q47558790 | Gene therapy for Mucopolysaccharidoses. |
| Q57167448 | Gene therapy for hemophilia: what does the future hold? |
| Q33881920 | Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects. |
| Q38607706 | Gene therapy returns to centre stage |
| Q26772109 | Gene therapy: progress and predictions |
| Q36290952 | Genome editing for inborn errors of metabolism: advancing towards the clinic. |
| Q36756903 | Genotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy? |
| Q38302375 | Helper-dependent adenoviral vectors for liver-directed gene therapy of primary hyperoxaluria type 1. |
| Q45875499 | Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant. |
| Q89777684 | Hemophilia gene therapy comes of age |
| Q45875635 | Hemophilia gene therapy comes of age. |
| Q90390133 | High levels of AAV vector integration into CRISPR-induced DNA breaks |
| Q90493905 | High-level protein production in erythroid cells derived from in vivo transduced hematopoietic stem cells |
| Q90402550 | ITR-Seq, a next-generation sequencing assay, identifies genome-wide DNA editing sites in vivo following adeno-associated viral vector-mediated genome editing |
| Q40219232 | In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model. |
| Q90211358 | In Vivo Delivery of Nucleic Acid-Encoded Monoclonal Antibodies |
| Q40875310 | Integration of AAV vectors and insertional mutagenesis |
| Q90001715 | Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency |
| Q41925391 | Liver-directed gene therapy for murine glycogen storage disease type Ib. |
| Q96591115 | Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B |
| Q91692021 | Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy |
| Q35199291 | Long-term sex-biased correction of circulating propionic acidemia disease markers by adeno-associated virus vectors |
| Q48555858 | Long-term sustained effect of liver-targeted AAV gene therapy for MNGIE. |
| Q37507816 | Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease |
| Q89509054 | Lysosomal Abnormalities in Cardiovascular Disease |
| Q54945184 | Methylmalonic acidemia: Current status and research priorities. |
| Q38957957 | Modeling correction of severe urea cycle defects in the growing murine liver using a hybrid recombinant adeno-associated virus/piggyBac transposase gene delivery system |
| Q100455120 | Modulating gene regulation to treat genetic disorders |
| Q52584002 | Molecular biology and gene therapy for glycogen storage disease type Ib. |
| Q40075764 | Neonatal Gene Therapy for Hemophilia B by a Novel Adenovirus Vector Showing Reduced Leaky Expression of Viral Genes |
| Q36825929 | Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic Acidemia |
| Q37698337 | Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease. |
| Q52429666 | Nuclease-Mediated Gene Therapies for Inherited Metabolic Diseases of the Liver. |
| Q38529941 | Obstacles and future of gene therapy for hemophilia |
| Q92345005 | Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease |
| Q98281054 | Plasmid DNA gene therapy of the Niemann-Pick C1 mouse with transferrin receptor-targeted Trojan horse liposomes |
| Q58804800 | Pompe disease: how to solve many problems with one solution |
| Q90269934 | Principles of Genetic Engineering |
| Q38601073 | Progress toward improved therapies for inborn errors of metabolism |
| Q41876346 | Promoterless gene targeting without nucleases rescues lethality of a Crigler-Najjar syndrome mouse model |
| Q38546492 | Recent advances in gene therapy for lysosomal storage disorders |
| Q37043497 | Recombinant AAV Integration Is Not Associated With Hepatic Genotoxicity in Nonhuman Primates and Patients |
| Q38907797 | Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models. |
| Q38970823 | Recombinant Antibody Fragments for Neurodegenerative Diseases |
| Q40613665 | Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas |
| Q40997212 | Reducible PEG-POD/DNA Nanoparticles for Gene Transfer In Vitro and In Vivo: Application in a Mouse Model of Age-Related Macular Degeneration |
| Q38531851 | Regulation of hepatocyte identity and quiescence. |
| Q40084021 | Repeated AAV-mediated gene transfer by serotype switching enables long-lasting therapeutic levels of hUgt1a1 enzyme in a mouse model of Crigler-Najjar Syndrome Type I. |
| Q42749763 | Reply to "Wild-type AAV Insertions in Hepatocellular Carcinoma Do Not Inform Debate Over Genotoxicity Risk of Vectorized AAV" |
| Q102075543 | Safety questions for AAV gene therapy |
| Q40581308 | Self-Complementary AAV9 Gene Delivery Partially Corrects Pathology Associated with Juvenile Neuronal Ceroid Lipofuscinosis (CLN3). |
| Q38809600 | Short DNA Hairpins Compromise Recombinant Adeno-Associated Virus Genome Homogeneity. |
| Q41964540 | Somatic genome editing with CRISPR/Cas9 generates and corrects a metabolic disease |
| Q36028204 | Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late-gestation nonhuman primates. |
| Q37577414 | TALEN/CRISPR-mediated engineering of a promoterless anti-viral RNAi hairpin into an endogenous miRNA locus |
| Q26772315 | Targeting adeno-associated virus and adenoviral gene therapy for hepatocellular carcinoma |
| Q64039743 | The Alpha-1-Antitrypsin Promoter Improves the Efficacy of an AAV Vector for the Treatment of MNGIE |
| Q90275396 | Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector-mediated intrauterine gene transfer in early-gestation fetal macaques |
| Q92861368 | Three-dimensional brain-like microenvironments facilitate the direct reprogramming of fibroblasts into therapeutic neurons |
| Q90595648 | Update on clinical gene therapy for hemophilia |
| Q38800643 | Viral Vectors: The Road to Reducing Genotoxicity |
| Q42749767 | Wild-type AAV Insertions in Hepatocellular Carcinoma Do Not Inform Debate Over Genotoxicity Risk of Vectorized AAV. |
| Q90350393 | ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome |
| Q60044774 | miR-122 removal in the liver activates imprinted microRNAs and enables more effective microRNA-mediated gene repression |
| Q42716243 | p62, Upregulated during Preneoplasia, Induces Hepatocellular Carcinogenesis by Maintaining Survival of Stressed HCC-Initiating Cells |
Search more.