About: In utero CRISPR-mediated therapeutic editing of metabolic genes

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description	article (en) wetenschappelijk artikel (nl) im Oktober 2018 veröffentlichter wissenschaftlicher Artikel (de) наукова стаття, опублікована в жовтні 2018 (uk)
publication date	wds:Q57296599-04AFB7FB-A860-4D76-AB5D-318FABF8EE7B
publication date	2018-10-08 00:00:00Z (xsd:dateTime)
language of work or name	wds:Q57296599-91D1EB92-F140-45DB-9B9F-6E08169FEA32
language of work or name	English
exact match	wds:Q57296599-BAAA70F9-676C-4644-8407-CD92F53F547F
exact match	https://scigraph.springernature.com/pub.10.1038/s41591-018-0184-6
cites work	wds:Q57296599-02124803-6CA9-48B1-A17C-B9F717E2BDD6 wds:Q57296599-09D75E46-ABEE-463B-91AC-89A93E6A953E wds:Q57296599-1021D271-6536-4956-9DA7-3C6F427D2AD6 wds:Q57296599-2926C035-45EE-4BA2-A17F-C1DE53117D4C wds:Q57296599-2CC4DE97-A0F3-4E4C-A51C-55B7643B8716 wds:Q57296599-384B440A-BF05-43B4-AA47-254F28ECE0B7 wds:Q57296599-4A16D28E-101A-4DB2-A06B-217D07C2EF3D wds:Q57296599-5F14E5B2-24F9-417A-ADEF-1FFE3B3247D7 wds:Q57296599-7DB17E83-76DD-43E3-9DD6-4253E1C0B2D3 wds:Q57296599-7E22D820-B32E-4EC2-B4C7-4BEF1EE2ADFD wds:Q57296599-87E94C17-B57E-4E39-AD12-6EA2846853F5 wds:Q57296599-948D405D-0AC9-4DB1-996F-806C17B4442B wds:Q57296599-99D86725-F042-46C6-B27F-F795FE2F1F45 wds:Q57296599-9BCA0FB4-6FB7-412E-819B-B22BEA1C19D8 wds:Q57296599-A7C33582-605A-41FD-A222-E3A51E440DA3 wds:Q57296599-B1100F3D-0563-4010-BFDF-BB5C75EBBEF4 wds:Q57296599-B6E6EB8D-8B8B-4A34-9724-C67C4EBD3DDF wds:Q57296599-B8A51FE6-B39E-40C1-AFDB-AB04EF5EF81A wds:Q57296599-BD84BB8B-0507-4285-A861-AC4C84D2ED79 wds:Q57296599-BE7C5451-E715-49EA-A976-C53FF2C1ADBE wds:Q57296599-D4FDD517-3A15-491A-9B2E-7FD8989BC22B wds:Q57296599-D65A1FE8-301A-4728-8AD8-595DA2CD8C94 wds:Q57296599-DA356F4F-2AFB-4213-A874-8A94C96A1B10 wds:Q57296599-DB07E89F-2C6B-4F2A-B7BD-3D23ACCF51B6 wds:Q57296599-E4B71A6E-2D3A-4449-AC01-F66EC1668927 wds:Q57296599-E5A5DB41-A4D1-40E7-A878-D82A1C5CBD8B wds:Q57296599-EC77A6BC-29F6-40EA-A613-C2E06C5D6469 wds:Q57296599-F55D08EB-0D70-408B-B42E-FD38ADE7E971 wds:Q57296599-F59014AD-9B91-4074-84BC-F2B5059A53E2 wds:Q57296599-F9B0F564-5F30-496B-AEBA-BFF524033A27 wds:Q57296599-FC737B26-B0D2-4BFD-A8BA-E0BBC8F12662
cites work	Point mutations in the murine fumarylacetoacetate hydrolase gene: Animal models for the human genetic disorder hereditary tyrosinemia type 1 Sequence variations in PCSK9, low LDL, and protection against coronary heart disease Adenovirus-Mediated Somatic Genome Editing of Pten by CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses. Permanent alteration of PCSK9 with in vivo CRISPR-Cas9 genome editing CRISPR-Cas9 Targeting of PCSK9 in Human Hepatocytes In Vivo-Brief Report Repeated Aerosolized AAV-CFTR for Treatment of Cystic Fibrosis: A Randomized Placebo-Controlled Phase 2B Trial Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype. In vivo genome editing using Staphylococcus aureus Cas9 In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor CD8(+) T-cell responses to adeno-associated virus capsid in humans Evaluation of off-target and on-target scoring algorithms and integration into the guide RNA selection tool CRISPOR. Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo Robust expansion of human hepatocytes in Fah-/-/Rag2-/-/Il2rg-/- mice Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo. Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice Reexpression following readministration of an adenoviral vector in adult mice after initial in utero adenoviral administration Adenoviral vector immunity: its implications and circumvention strategies Persistent expression of hF.IX After tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia Genome-wide target specificities of CRISPR RNA-guided programmable deaminases The developmental stage determines the distribution and duration of gene expression after early intra-amniotic gene transfer using lentiviral vectors Increasing the genome-targeting scope and precision of base editing with engineered Cas9-cytidine deaminase fusions In Vivo Base Editing of PCSK9 (Proprotein Convertase Subtilisin/Kexin Type 9) as a Therapeutic Alternative to Genome Editing The Impact of Chromatin Dynamics on Cas9-Mediated Genome Editing in Human Cells Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing. A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing. The Intravenous Route of Injection Optimizes Engraftment and Survival in the Murine Model of In Utero Hematopoietic Cell Transplantation. Induction of Immune Tolerance to Foreign Protein via Adeno-Associated Viral Vector Gene Transfer in Mid-Gestation Fetal Sheep Biochemical and Clinical Aspects of Hereditary Tyrosinemia Type 1. Prenatal Diagnosis of Tyrosinemia Type 1 Using Next Generation Sequencing.
author name string	wds:Q57296599-12AA0C8A-A1A9-4977-B4B8-A427A2418F98 wds:Q57296599-3837BA23-07F8-441F-8C5A-957937F947A4 wds:Q57296599-48E2BF58-9D54-4B02-B6A4-A72ECA66E4B5 wds:Q57296599-4957D335-CCAC-4977-B82E-7D2E010E1EBB wds:Q57296599-4E6B7A90-E6BD-4FDE-9A12-9B8BE8B55EB0 wds:Q57296599-6A2DD7BE-B5A2-4CAC-8650-23BB6F3DC6BD wds:Q57296599-8B10BC60-7EF9-4831-9FD2-09149EA04E50 wds:Q57296599-9258DF41-25AD-45C6-A6C2-F1D75BC51988 wds:Q57296599-93B87036-CEC3-4BD0-85A8-4BAAB30E8F88 wds:Q57296599-AED8305C-C380-4472-9B31-3F39A2CD4059 wds:Q57296599-B58D45A4-D467-4CF3-AAC9-11391CBFB855 wds:Q57296599-D1E89251-F215-4FEE-B6C8-551F7F6C05E3 wds:Q57296599-DEF9D3C2-151D-4C07-BA37-BA49ECCD9AB0 wds:Q57296599-E703720E-3B8E-4505-A040-B0C22654D5AF wds:Q57296599-EBD37D92-7E60-4C11-9A23-3E38AA8027ED
author name string	Li Li Haiying Li Rajan Jain Kiran Musunuru Edward E Morrisey Philip W Zoltick William H Peranteau Kshitiz Singh Alexandra C Chadwick Avery C Rossidis John D Stratigis Wenjian Lv

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