Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery. - Wikidata - awgldk - TriplyDB

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.

http://www.wikidata.org/entity/Q36187202

about

Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials Dystrophin-deficient large animal models: translational research and exon skipping A chemical view of oligonucleotides for exon skipping and related drug applications Splicing therapy for neuromuscular disease Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy The mdx mouse model as a surrogate for Duchenne muscular dystrophy The Dystrophin Complex: Structure, Function, and Implications for Therapy Parathyroid hormone and parathyroid hormone type-1 receptor accelerate myocyte differentiation.Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy.Human skeletal muscle xenograft as a new preclinical model for muscle disorders Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders.Pharmacologic management of Duchenne muscular dystrophy: target identification and preclinical trials Exon skipping restores dystrophin expression, but fails to prevent disease progression in later stage dystrophic dko mice Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.Antisense therapy in neurology Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy.Targeted excision of VCP R155H mutation by Cre-LoxP technology as a promising therapeutic strategy for valosin-containing protein disease.Long-term efficacy of systemic multiexon skipping targeting dystrophin exons 45-55 with a cocktail of vivo-morpholinos in mdx52 mice Exon-skipping therapy: a roadblock, detour, or bump in the road?Duchenne muscular dystrophy: current cell therapies X-Linked Dilated Cardiomyopathy: A Cardiospecific Phenotype of Dystrophinopathy.Non-sequential and multi-step splicing of the dystrophin transcript.Autonomic Modulation in Duchenne Muscular Dystrophy during a Computer Task: A Prospective Control Trial.Mutation types and aging differently affect revertant fiber expansion in dystrophic mdx and mdx52 mice.Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy.Development of multiexon skipping antisense oligonucleotide therapy for Duchenne muscular dystrophy Splice-switching antisense oligonucleotides as therapeutic drugs Endogenous Multiple Exon Skipping and Back-Splicing at the DMD Mutation Hotspot.Morpholino treatment improves muscle function and pathology of Pitx1 transgenic mice.Genome Editing Gene Therapy for Duchenne Muscular Dystrophy.Current Translational Research and Murine Models For Duchenne Muscular Dystrophy.Enhancement of Satellite Cell Transplantation Efficiency by Leukemia Inhibitory Factor.Repair or replace? Exploiting novel gene and cell therapy strategies for muscular dystrophies.Ongoing therapeutic trials and outcome measures for Duchenne muscular dystrophy.New developments in the use of gene therapy to treat Duchenne muscular dystrophy.Oligonucleotide-Based Therapy for FTD/ALS Caused by the C9orf72 Repeat Expansion: A Perspective.New developments in exon skipping and splice modulation therapies for neuromuscular diseases.What has the mdx mouse model of Duchenne muscular dystrophy contributed to our understanding of this disease?Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy.Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice.

P2860

Q26741409-69CEEC96-CC65-4F38-8F24-B1BEE6379E57 Q26781901-0DC0FE48-D82E-4A9F-93E0-E2B4EFAEE79E Q26863684-879B09F9-114E-4E7A-AC27-31F9D67479E4 Q27009581-55CC9D49-9915-4E22-8FAC-8474A62022AC Q28084979-57C04970-D3CC-42E1-BF08-1DCF98FE4BD1 Q28656377-F19D5107-8C37-4833-95E1-FCE122DCC41F Q30376293-7D886D0B-A0AB-4310-B115-388A8DDEF4E1 Q30580472-F9E11A26-7DA3-4728-A11C-E91627231FA3 Q33594805-6BA0B2C8-D651-4DCD-AB69-36A39A0807EA Q33649287-FD795AC3-AA79-4097-9739-C47664617F59 Q34045672-6778D576-E1B1-4016-964A-6D4FE7FCE56A Q34152280-FE79C4FD-FC90-4862-9C4F-B6F929EF4241 Q34204198-23D7033B-BD02-4633-8560-7AD1904C0122 Q34463483-C1D2F124-F304-4F68-B4AB-4BB03A8E437F Q34608717-F50D2F32-701B-421A-8941-2662ABD9842B Q34883011-9FF49BA7-CA30-4FB3-B6B1-230D9BC71B81 Q35110776-103FB5EF-2BF4-4F04-880E-CFFC67A3EC2B Q35133936-6841608C-367A-4284-92F4-5AA71397503A Q35731229-CD7AEAAE-C9AA-4212-8873-ADAC4EC4F7CE Q35780806-A62E8EE8-415C-44DC-86A6-7DB914FDC1D0 Q35818371-C9E5004F-FF11-412D-A986-F8B00038C768 Q35869261-C27D4FD0-4C65-4DCD-8B62-1DAFEEB0447A Q36258747-64CC2DEE-5CD4-4E82-9AC8-A68B748BC53C Q37042659-F0A36439-F712-46CE-981E-575CBCA4CC05 Q37053612-7505C500-B5BA-4344-A459-BE93B558AF6A Q37105210-68D24D10-9188-4922-BF61-78A566F4E92B Q37211004-491B06D8-27E8-4F26-A107-8430161FB24C Q37377488-B4CBA69C-BE8A-4AEF-BB1E-9339BE06D69D Q37562102-E52C5191-24D6-4A01-9F74-D7F80616AA67 Q37589752-15C1E62A-496F-4A1C-AB98-6B41968E1EE2 Q37610505-1B177924-08A9-4D98-BCDF-F2ACAC33DEE3 Q37610588-1DFDF2E8-6250-4822-B873-350691F6003A Q38079633-E85DB754-D3DF-4D94-98BC-F7A24E2EE39C Q38115148-107F0C20-C72A-4640-B63F-B1E8F436DD14 Q38168863-FB6924F5-0D52-4F6A-B3B7-BF1D13DEACE2 Q38172172-B28B99AD-EFA5-46C9-9125-37F0C1AC94D7 Q38195295-FD0902D5-3FBC-40D9-BC50-00073F67C4C3 Q38351283-D62AAD69-010C-4629-B116-59257277B870 Q38829627-A2EBF88E-6ECE-4C5A-9CE6-24A883FC117D Q39120926-9CD42CD1-EC36-4F1B-AEE6-F082FB92FE0D

P2860

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.

http://www.wikidata.org/entity/Q36187202

description

2012 nî lūn-bûn

@nan

2012年の論文

@ja

2012年論文

@yue

2012年論文

@zh-hant

2012年論文

@zh-hk

2012年論文

@zh-mo

2012年論文

@zh-tw

2012年论文

@wuu

2012年论文

@zh

2012年论文

@zh-cn

name

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.

@ast

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.

@en

type

label

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.

@ast

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.

@en

prefLabel

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.

@ast

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.

@en

P1433

Q36187202-F463042D-95FF-41B2-A063-EB067AED7603

P1476

Q36187202-0AA676E4-32B0-4BAC-92C4-A215AA3849AC

P2093

Q36187202-012993A9-4378-4CF3-B42D-1B2E08A24B3E

Q36187202-1BF63F76-A026-4881-AD55-1D51FBBCD27B

Q36187202-22FD6ED4-0D4F-42DC-A2E4-114EE07F68C8

Q36187202-5325A988-5D0F-40AF-A6EE-31AA8E0FBE30

Q36187202-6C5F9939-C9F4-4E0D-82AD-81C4506EAC5C

Q36187202-74EE1E65-9867-467D-8DF7-CD6E8966730B

Q36187202-9E4157AF-61F4-4F84-AC5A-53698A436941

Q36187202-C6FC61FE-BB69-457E-9478-9E30766506BC

Q36187202-D7C3D526-B1C7-4D83-ABF9-30D9F9AF449F

P2860

Q36187202-0E7A0A11-24BF-48D8-AB2A-6A8B7C967AFC

Q36187202-14E0CEFF-DF8E-4DD8-B4AE-1E681E0BDC72

Q36187202-3B9386A4-6556-407F-A245-1F11400506A5

Q36187202-3E7945AE-F6DA-48AD-8B63-DD3C43DC1CD5

Q36187202-4459C533-F793-4FFD-B57A-F7C33A55638F

Q36187202-476D044F-4452-4A46-854F-FCFE72DEF087

Q36187202-493995CC-4582-45D7-9D9D-A1DEA8C2720A

Q36187202-6471F219-AA7E-44B1-85C9-B0573887DF4B

Q36187202-64D5F34C-9506-40B8-AC30-58A5AC493BFC

Q36187202-817A8925-95D3-4D2B-B824-4B2F6A205D33

P304

Q36187202-B102FF97-0E18-4048-8342-55B56B25F829

P31

Q36187202-EFFF597B-C5E6-41C1-A5E5-518DAFF182EF

P356

Q36187202-3B473D71-5326-48D8-A54F-68CC2C8A6698

P407

Q36187202-78069197-B728-4298-A3D8-4302BF8229F3

P433

Q36187202-18B2EAA9-0118-45AB-BEFA-9DF7B4FBE007

P478

Q36187202-E8A1D32E-BE15-42E7-ABE6-46BEDB937670

P50

Q36187202-0B4F480D-30D3-4BEC-B8C5-B50394861F21

Q36187202-181836F2-1F72-4868-9B83-302F94598DB9

P577

Q36187202-EE808396-2C12-4438-A42C-F624BCDF3B32

P5875

Q36187202-DCBE070D-00BD-4F9C-B37A-B49310663F21

P698

Q36187202-71CBA646-C5D9-4ADF-AC00-5FDB317A9C8C

P932

Q36187202-0A764C67-CCCB-4504-8617-530DD03DEBF2

P356

PNAS.1204638109

P1433

Proceedings of the National Academy of Sciences of the United States of America

P1476

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.

@en

P2093

Akinori Nakamura

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

Eric P Hoffman

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

Jun Tanihata

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

Shin'ichi Takeda

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

Takashi Saito

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

Terence Partridge

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

Tetsuya Nagata

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

Toshifumi Yokota

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

Yoshitsugu Aoki

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

P2860

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

IDT SciTools: a suite for analysis and design of nucleic acid oligomers

Morpholino antisense oligomers: design, preparation, and properties

Antisense PMO found in dystrophic dog model was effective in cells from exon 7-deleted DMD patient

ESEfinder: A web resource to identify exonic splicing enhancers

Dystrophin: the protein product of the Duchenne muscular dystrophy locus

Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy

Assessment of the feasibility of exon 45-55 multiexon skipping for Duchenne muscular dystrophy

Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles

A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new ground

P304

13763-13768

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

P31

scholarly article

P356

10.1073/PNAS.1204638109

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

P407

P433

34

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

P478

109

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

P50

Kanneboyina Nagaraju

Stephanie Duguez

P577

2012-08-06T00:00:00Z

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

P5875

230624013

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

P698

22869723

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

P932

3427064

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