In vivo genome editing of the albumin locus as a platform for protein replacement therapy - Wikidata - wikimedia - TriplyDB

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

http://www.wikidata.org/entity/Q36143241

about

Genome Engineering with TALE and CRISPR Systems in Neuroscience Genome-editing Technologies for Gene and Cell Therapy Towards a new era in medicine: therapeutic genome editing Genome-editing technologies for gene correction of hemophilia Hematopoietic Stem Cells in Regenerative Medicine: Astray or on the Path?Long-Term Assessment of AAV-Mediated Zinc Finger Nuclease Expression in the Mouse Brain.Genome-Edited T Cell Therapies.CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice.Gene therapy for lysosomal storage disorders: a good start.Genome editing for inborn errors of metabolism: advancing towards the clinic.Gene Therapy: The View from NCATS.A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice.Adeno-associated viral vectors for the treatment of hemophilia.A genome editing primer for the hematologist A CRISPR/Cas9 Functional Screen Identifies Rare Tumor Suppressors.CRISPR-Cas9: a promising tool for gene editing on induced pluripotent stem cells.TALEN/CRISPR-mediated engineering of a promoterless anti-viral RNAi hairpin into an endogenous miRNA locus.From hacking the human genome to editing organs Creating and evaluating accurate CRISPR-Cas9 scalpels for genomic surgery.Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives.Current Progress in Therapeutic Gene Editing for Monogenic Diseases Salient Features of Endonuclease Platforms for Therapeutic Genome Editing Gene editing technology as an approach to the treatment of liver diseases.Therapeutic genome engineering via CRISPR-Cas systems.Programmable Site-Specific Nucleases for Targeted Genome Engineering in Higher Eukaryotes.CRISPR/Cas9-mediated somatic and germline gene correction to restore hemostasis in hemophilia B mice.Specific factor IX mRNA and protein features favor drug-induced readthrough over recurrent nonsense mutations.Delivery technologies for genome editing.Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications.Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I.Next-generation AAV vectors for clinical use: an ever-accelerating race.Gene Therapy in Tyrosinemia: Potential and Pitfalls.Corporate profile: Sangamo BioSciences, Inc.Promoterless gene targeting without nucleases rescues lethality of a Crigler-Najjar syndrome mouse model.A Perspective on the State of Genome Editing.Phase I and II clinical trials for the mucopolysaccharidoses.Presentation and Treatments for Mucopolysaccharidosis Type II (MPS II; Hunter Syndrome).The Future of CRISPR Applications in the Lab, the Clinic and Society.NOD2 and bacterial recognition as therapeutic targets for Crohn's disease.CRISPR/Cas9: A Potential Life-Saving Tool. What's next?

P2860

Q26752488-61032770-AF6F-48DD-8C5B-42C02F7B5642 Q26769662-EB98CFDA-D3C2-48F1-8BDD-8D1881375562 Q26773287-B537F49B-CCFF-4D5A-B5AE-CF450E63EC83 Q28066273-E30A24E8-D0F5-4D70-BB0E-807E2BCA9156 Q28072887-1020DF91-9A11-438D-AFBA-8442E9B8083A Q33715367-714BCD3D-21B7-4D11-99A4-C251A054BBE0 Q33729598-BC565FE8-73C1-41ED-AE34-29C917344D37 Q33830117-8BDB9D05-8936-499C-92E4-F63D346C15B5 Q34502978-3CC0CD24-F0FB-44D4-B8DF-C441C9DF7595 Q36290952-707EB1D0-953D-4FA7-ABB1-D385F1B2C55E Q36551233-A865008D-416B-47A3-BE0F-FC46B882FE8F Q36673956-078AABC8-5DBF-4D56-9C8F-947F65FDD562 Q36713767-4098B648-85AB-4DB4-85FC-34C0351B9770 Q36942027-7C0A54F8-DEE1-4289-966C-B6DF62D23E39 Q37508613-B98DC1D8-D3F2-41A7-A25C-E2D9871DBBED Q37559174-A55C531D-2C4B-40BE-9F3E-C5143D099958 Q37577414-73A797E7-967F-4483-AF6A-A5A677E08F8A Q38640256-4E9CF74A-B085-448C-8907-6C99A74E13BC Q38682882-7D8AA343-93BF-43E0-BD14-885FB6768698 Q38684097-1BF08DD7-3F92-4B82-9A7F-7A14505F95E7 Q38696285-C91323EC-7A78-4B14-9157-4F6472F63484 Q38706622-B8D7D780-EDAF-4B92-A5E1-A269BE143A6B Q38749884-B7C7BA6F-0711-47B6-9D70-B2C7B7175819 Q38757432-F0996BBB-9DB1-4868-AC0B-5822FA095A58 Q38761853-8BEE8C9D-7487-4663-893C-2304B0647573 Q38784208-BE79AFF2-B594-4FFB-AE23-E7453FA97FB1 Q38961683-666DE930-19A5-4C92-8D2A-3ACA456942B7 Q39197557-F06014E7-3573-48D9-8438-703D4D633AFD Q39227498-7E0164E8-7816-4C14-89CF-29FEE0E53CCE Q39339697-9D5DA03B-EF88-411C-B3B0-A800F3079C9E Q40100060-83E67341-E115-4E07-927E-5C55C2DAA26D Q40103301-B6FFD206-4771-43A2-B048-EAA1CCEA3171 Q40659374-C385984F-70E4-4D91-B754-FE5696CC866C Q41876346-B493B393-7E7B-41A2-8970-39C077ADB12E Q42546298-22AB9EF6-3A95-423D-8E34-4DD2676E4727 Q45873551-B8B99DA7-8B13-46F7-85EA-9CCAEC4458F7 Q45874778-4F65F23F-BC2E-4247-BF18-DE013D275B9E Q46262974-86245097-27E6-439C-A7A9-4CF3B7CEEEB9 Q46269946-853CAA19-F7DC-4CB5-84BE-244BA8B6CA0B Q47095305-67475E17-36E0-44E6-BF2D-ED964B42D815

P2860

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

http://www.wikidata.org/entity/Q36143241

description

2015 nî lūn-bûn

@nan

2015年の論文

@ja

2015年論文

@yue

2015年論文

@zh-hant

2015年論文

@zh-hk

2015年論文

@zh-mo

2015年論文

@zh-tw

2015年论文

@wuu

2015年论文

@zh

2015年论文

@zh-cn

name

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

@ast

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

@en

type

label

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

@ast

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

@en

prefLabel

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

@ast

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

@en

P1433

Q36143241-F6EB3DE7-4C66-4F00-96C5-BC9012154BAD

P1476

Q36143241-27CEF272-D38E-457E-957F-0D9E517C4F87

P2093

Q36143241-26ADB0F7-74FE-4034-8F0F-E8A2F5EB9449

Q36143241-2744ABF2-DFC6-4FB8-9481-309936011137

Q36143241-4FCB2CEB-FDC6-43C9-8849-654DB03DF7FE

Q36143241-539730E0-437D-4ED0-8D7E-A7ECC37BD4D6

Q36143241-56AB051E-3FD9-4E7D-9C68-D6C81625F704

Q36143241-735F9689-95A8-4140-BDF1-54FE5AFC1013

Q36143241-79672BE5-4070-4D6F-BB5C-FF0B0BC3E4F1

Q36143241-8655E5EB-0BA3-46E9-830A-795EFB4B74BB

Q36143241-C3BD4ECD-1591-4B52-BCB4-2DCDD8EBEFE8

Q36143241-E5E81E75-1273-4DC5-B5F2-8D33FF8E7B1B

P2860

Q36143241-09BB69F6-87C9-498B-B3B7-6F7ADFCF2F73

Q36143241-15A715F7-B4A2-4CCE-AAE8-1119CB5B832A

Q36143241-181B6DBE-5AC5-4B64-8A9B-9BA7DC65ACF3

Q36143241-1ACA39EB-726E-4721-8D3B-7C5DD3E3C24F

Q36143241-2569B39F-8C4B-4BA4-98A1-4D1DA2EAC751

Q36143241-288980CE-8D5C-4D1F-A57E-33780B98F246

Q36143241-305480DC-3F8E-4F5A-AE83-C475D2B6A200

Q36143241-4244905D-1263-4FAE-A7A6-1ADD092B2C86

Q36143241-44F96206-DDB0-4172-BB21-6D657D728CFE

Q36143241-4D7B2D5B-DA31-4319-ADE9-D5EBE0E4A2C4

P304

Q36143241-09CEB48F-1155-4959-8BA3-FE218F5A944E

P31

Q36143241-4B2AA9A0-C51F-4752-9279-E15A2117DD26

P356

Q36143241-701071DB-A580-4CAB-A3C8-662F46703620

P407

Q36143241-EC010CD5-E721-461F-8D77-AF17333FE0B8

P433

Q36143241-7C53F0BC-2FCE-44C1-A32B-36F2B36060FD

P478

Q36143241-549C22F7-E2EC-415B-9D42-E5F3B9AB5039

P50

Q36143241-2FA02FB9-7167-4FCB-9DF6-AF2376FF4A83

Q36143241-3E4C020D-C845-452B-9225-925CDE802517

Q36143241-9F731492-4C6B-48F3-A49B-E6CFE73F139F

Q36143241-D686D6DD-90E8-454A-8550-EB89D2A0FE79

Q36143241-ED7B28D7-CEFB-4767-84FC-7352985D1273

P577

Q36143241-E97361F7-B2CA-4582-807F-B1E9F7F0B9A0

P698

Q36143241-61D42E42-1C6D-49DA-8046-11C6A0498708

P932

Q36143241-F3BC63E7-A37F-4EFC-B78A-4768B15C819E

P356

BLOOD-2014-12-615492

P1433

P1476

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

@en

P2093

David E Paschon

http://www.w3.org/2001/XMLSchema#string

David Shivak

http://www.w3.org/2001/XMLSchema#string

Edward J Rebar

http://www.w3.org/2001/XMLSchema#string

Jeffrey C Miller

http://www.w3.org/2001/XMLSchema#string

Julianne Rieders

http://www.w3.org/2001/XMLSchema#string

Michael C Holmes

http://www.w3.org/2001/XMLSchema#string

Robert J Davidson

http://www.w3.org/2001/XMLSchema#string

Russell C DeKelver

http://www.w3.org/2001/XMLSchema#string

Scott Sproul

http://www.w3.org/2001/XMLSchema#string

Shangzhen Zhou

http://www.w3.org/2001/XMLSchema#string

P2860

Analysis of Relative Gene Expression Data Using Real-Time Quantitative PCR and the 2−ΔΔCT Method

Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV

In vivo genome editing restores haemostasis in a mouse model of haemophilia

Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection

Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures

Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases

AAV2 gene therapy readministration in three adults with congenital blindness.

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype.

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

P304

1777-1784

http://www.w3.org/2001/XMLSchema#string

P31

scholarly article

P356

10.1182/BLOOD-2014-12-615492

http://www.w3.org/2001/XMLSchema#string

P407

P433

15

http://www.w3.org/2001/XMLSchema#string

P478

126

http://www.w3.org/2001/XMLSchema#string

P50

Philip D Gregory

Xavier M Anguela

Katherine A High

P577

2015-08-21T00:00:00Z

http://www.w3.org/2001/XMLSchema#dateTime

P698

26297739

http://www.w3.org/2001/XMLSchema#string

P932

4600017

http://www.w3.org/2001/XMLSchema#string