| scholarly article | Q13442814 |
| P6179 | Dimensions Publication ID | 1039253377 |
| P356 | DOI | 10.1038/NATURE13864 |
| P932 | PMC publication ID | 4297598 |
| P698 | PubMed publication ID | 25363772 |
| P5875 | ResearchGate publication ID | 267753151 |
| P50 | author | Paul N. Valdmanis | Q54282802 |
| Nicole K. Paulk | Q55206953 | ||
| P2093 | author name string | Y Huang | |
| Y Shi | |||
| F Zhang | |||
| M A Kay | |||
| K Chu | |||
| M H Porteus | |||
| K M Gaensler | |||
| A Barzel | |||
| L P Spector | |||
| P2860 | cites work | Gene therapy with human and mouse T-cell receptors mediates cancer regression and targets normal tissues expressing cognate antigen | Q24623215 |
| In vivo genome editing restores haemostasis in a mouse model of haemophilia | Q28241725 | ||
| Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1 | Q28290118 | ||
| Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial. | Q28306059 | ||
| High cleavage efficiency of a 2A peptide derived from porcine teschovirus-1 in human cell lines, zebrafish and mice | Q28740624 | ||
| ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering | Q29615505 | ||
| High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells | Q29616045 | ||
| In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses | Q33327474 | ||
| Quantifying genome-editing outcomes at endogenous loci with SMRT sequencing | Q33587979 | ||
| Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo | Q33844362 | ||
| Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype | Q34233154 | ||
| Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. | Q34356904 | ||
| Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways | Q34530552 | ||
| Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo. | Q35105022 | ||
| Antibody-based protection against HIV infection by vectored immunoprophylaxis | Q35658856 | ||
| Adenovirus-associated virus vector-mediated gene transfer in hemophilia B | Q35691659 | ||
| AAV-mediated gene targeting is significantly enhanced by transient inhibition of nonhomologous end joining or the proteasome in vivo | Q36084471 | ||
| Single amino acid modification of adeno-associated virus capsid changes transduction and humoral immune profiles | Q36171820 | ||
| Ribosomal DNA integrating rAAV-rDNA vectors allow for stable transgene expression. | Q36298655 | ||
| A mini-intronic plasmid (MIP): a novel robust transgene expression vector in vivo and in vitro | Q36883864 | ||
| Role of antigen-specific regulatory CD4+CD25+ T cells in tolerance induction after neonatal IP administration of AAV-hF.IX. | Q37226788 | ||
| Humoral Immune Response to AAV. | Q37236099 | ||
| Robust ZFN-mediated genome editing in adult hemophilic mice. | Q37291607 | ||
| Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases. | Q37420333 | ||
| Selection and evaluation of clinically relevant AAV variants in a xenograft liver model | Q37610916 | ||
| Organogenesis and development of the liver | Q37693447 | ||
| Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice | Q37747148 | ||
| Mechanisms of retroviral integration and mutagenesis | Q38075040 | ||
| Albumin: pathophysiologic basis of its role in the treatment of cirrhosis and its complications | Q38083035 | ||
| Optimal Immunofluorescent Staining for Human Factor IX and Infiltrating T Cells following Gene Therapy for Hemophilia B. | Q42092752 | ||
| In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous | Q45350780 | ||
| Gene targeting in vivo by adeno-associated virus vectors. | Q45413871 | ||
| AAV vector integration sites in mouse hepatocellular carcinoma. | Q45869780 | ||
| A reproducible and well-tolerated method for 2/3 partial hepatectomy in mice | Q47564825 | ||
| Gene therapy for lysosomal storage disorders | Q95501632 | ||
| P433 | issue | 7534 | |
| P407 | language of work or name | English | Q1860 |
| P921 | main subject | hemophilia | Q134003 |
| gene targeting | Q211083 | ||
| hemophilia B | Q2562598 | ||
| P304 | page(s) | 360-364 | |
| P577 | publication date | 2014-10-29 | |
| P1433 | published in | Nature | Q180445 |
| P1476 | title | Promoterless gene targeting without nucleases ameliorates haemophilia B in mice | |
| P478 | volume | 517 |
| Q91826241 | A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I |
| Q36673956 | A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice |
| Q36942027 | A genome editing primer for the hematologist |
| Q89869568 | A mutation-independent CRISPR-Cas9-mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiency |
| Q37313599 | A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome |
| 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 |
| Q90181593 | AAV6 Is Superior to Clade F AAVs in Stimulating Homologous Recombination-Based Genome Editing in Human HSPCs |
| Q42584196 | Adeno-associated virus finds its disease |
| Q64040883 | Adeno-associated virus vector as a platform for gene therapy delivery |
| Q42565179 | An Orthogonal Array Optimization of Lipid-like Nanoparticles for mRNA Delivery in Vivo. |
| Q45873427 | Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity. |
| Q36849517 | CRISPR-Cas9 Targeting of PCSK9 in Human Hepatocytes In Vivo-Brief Report |
| Q64059247 | CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells |
| Q98292026 | CRISPR-Cas9-Mediated In Vivo Gene Integration at the Albumin Locus Recovers Hemostasis in Neonatal and Adult Hemophilia B Mice |
| Q40116681 | CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors |
| Q48166206 | CRISPR/Cas9 genome editing in human hematopoietic stem cells |
| Q40486239 | CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells |
| Q37446641 | CRISPR/Cas9-mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse |
| Q26743393 | Clinical development of gene therapy: results and lessons from recent successes |
| Q92845907 | Coupling AAV-mediated promoterless gene targeting to SaCas9 nuclease to efficiently correct liver metabolic diseases |
| Q35986193 | Cre Activated and Inactivated Recombinant Adeno-Associated Viral Vectors for Neuronal Anatomical Tracing or Activity Manipulation |
| Q92017672 | Curing hemophilia A by NHEJ-mediated ectopic F8 insertion in the mouse |
| Q38696285 | Current Progress in Therapeutic Gene Editing for Monogenic Diseases |
| Q38827606 | Current and future prospects for hemophilia gene therapy |
| Q64881813 | Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing. |
| Q89624120 | Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes |
| Q47554736 | Emerging Issues in AAV-Mediated In Vivo Gene Therapy |
| Q102141457 | Engineered B cells expressing an anti-HIV antibody enable memory retention, isotype switching and clonal expansion |
| Q89777435 | Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors |
| Q92239257 | Episomal vectors based on S/MAR and the β-globin Replicator, encoding a synthetic transcriptional activator, mediate efficient γ-globin activation in haematopoietic cells |
| Q98164401 | Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins |
| Q38772195 | Future of rAAV Gene Therapy: Platform for RNAi, Gene Editing, and Beyond |
| Q38729963 | Gene Insertion Into Genomic Safe Harbors for Human Gene Therapy |
| Q51057724 | Gene Therapy for Hemophilia. |
| Q92219605 | Gene and Base Editing as a Therapeutic Option for Cystic Fibrosis-Learning from Other Diseases |
| Q38749884 | Gene editing technology as an approach to the treatment of liver diseases |
| Q90383791 | Gene therapy and genome editing for primary immunodeficiency diseases |
| Q28087557 | Gene therapy in an era of emerging treatment options for hemophilia B |
| Q38607706 | Gene therapy returns to centre stage |
| Q41087426 | Genome Editing of the Blood: Opportunities and Challenges |
| Q36675277 | Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research Applications |
| Q36290952 | Genome editing for inborn errors of metabolism: advancing towards the clinic. |
| Q91654872 | Genome editing technologies to treat rare liver diseases |
| Q26769662 | Genome-editing Technologies for Gene and Cell Therapy |
| Q28066273 | Genome-editing technologies for gene correction of hemophilia |
| Q45873930 | Hematopoietic Stem Cell Gene Therapy: Progress and Lessons Learned |
| Q35591457 | Hitting the target without pulling the trigger |
| Q90196148 | Homologous Recombination-Based Genome Editing by Clade F AAVs Is Inefficient in the Absence of a Targeted DNA Break |
| Q36828138 | Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors. |
| Q38761267 | Human somatic cells deficient for RAD52 are impaired for viral integration and compromised for most aspects of homology-directed repair |
| Q38812436 | Improved methods of AAV-mediated gene targeting for human cell lines using ribosome-skipping 2A peptide |
| Q36951279 | In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA. |
| Q38804017 | In vivo blunt-end cloning through CRISPR/Cas9-facilitated non-homologous end-joining |
| Q36143241 | In vivo genome editing of the albumin locus as a platform for protein replacement therapy |
| Q52733586 | Liver tissue engineering: From implantable tissue to whole organ engineering. |
| Q28085748 | Liver-targeted gene therapy: Approaches and challenges |
| Q64374981 | Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9 |
| Q91692021 | Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy |
| Q38700095 | Mechanistic basis for increased human gene targeting by promoterless vectors-roles of homology arms and Rad54 paralogs. |
| Q59357423 | Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol |
| Q91670589 | Molecular Design, Optimization, and Genomic Integration of Chimeric B Cell Receptors in Murine B Cells |
| Q92989928 | Molecular Mechanisms and Determinants of Innovative Correction Approaches in Coagulation Factor Deficiencies |
| Q47141518 | Multiplexed in vivo homology-directed repair and tumor barcoding enables parallel quantification of Kras variant oncogenicity. |
| Q38622440 | New and improved AAVenues: current status of hemophilia B gene therapy |
| Q38543280 | New approaches to gene and cell therapy for hemophilia. |
| Q40100060 | Next-generation AAV vectors for clinical use: an ever-accelerating race |
| Q99580017 | Novel genetic therapeutic approaches for modulating the severity of β-thalassemia (Review) |
| Q59349832 | Nuclease-free Adeno-Associated Virus-Mediated Il2rg Gene Editing in X-SCID Mice |
| Q38529941 | Obstacles and future of gene therapy for hemophilia |
| Q45873054 | Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1. |
| Q41876346 | Promoterless gene targeting without nucleases rescues lethality of a Crigler-Najjar syndrome mouse model |
| Q61811175 | Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy |
| Q37279525 | Recombinant adeno-associated virus vectors in the treatment of rare diseases |
| Q35146211 | Regenerative medicine: targeted genome editing in vivo |
| Q48507292 | Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression. |
| Q94487328 | Split AAV-Mediated Gene Therapy Restores Ureagenesis in a Murine Model of Carbamoyl Phosphate Synthetase 1 Deficiency |
| Q59356657 | Stem cell-derived clade F AAVs mediate high-efficiency homologous recombination-based genome editing |
| Q37458718 | Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to success. |
| Q37577414 | TALEN/CRISPR-mediated engineering of a promoterless anti-viral RNAi hairpin into an endogenous miRNA locus |
| Q52431082 | Targeted in vivo knock-in of human alpha-1-antitrypsin cDNA using adenoviral delivery of CRISPR/Cas9. |
| Q33652566 | Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo |
| Q28087380 | Therapeutic genome editing: prospects and challenges |
| Q50956123 | To cleave or not to cleave: therapeutic gene editing with and without programmable nucleases. |
| Q26773287 | Towards a new era in medicine: therapeutic genome editing |
| Q41532325 | Transient suppression of hepatocellular replication in the mouse liver following transduction with recombinant adeno-associated virus. |
| Q30381138 | Ultrasound-targeted hepatic delivery of factor IX in hemophiliac mice. |
| Q90595648 | Update on clinical gene therapy for hemophilia |
| Q35056714 | Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy. |
| Q64375090 | Viral Vector-Based Delivery of CRISPR/Cas9 and Donor DNA for Homology-Directed Repair in an In Vitro Model for Canine Hemophilia B |
| Q38800643 | Viral Vectors: The Road to Reducing Genotoxicity |
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| Q85963380 | [Research advances on gene therapy for hemophilia A] |
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