Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer - Wikidata - awgldk - TriplyDB

Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer

Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer

http://www.wikidata.org/entity/Q30498316

about

AAV-mediated liver-specific MPV17 expression restores mtDNA levels and prevents diet-induced liver failure Therapies for the bone in mucopolysaccharidoses Impact of age at administration, lysosomal storage, and transgene regulatory elements on AAV2/8-mediated rat liver transduction Neonatal gene therapy with a gamma retroviral vector in mucopolysaccharidosis VI cats.Sensory-motor behavioral characterization of an animal model of Maroteaux-Lamy syndrome (or Mucopolysaccharidosis VI).Similar therapeutic efficacy between a single administration of gene therapy and multiple administrations of recombinant enzyme in a mouse model of lysosomal storage disease Gene transfer to the CNS using recombinant adeno-associated virus Therapies of mucopolysaccharidosis IVA (Morquio A syndrome).Neonatal bone marrow transplantation prevents bone pathology in a mouse model of mucopolysaccharidosis type I.Prevalence of anti-adeno-associated virus serotype 8 neutralizing antibodies and arylsulfatase B cross-reactive immunologic material in mucopolysaccharidosis VI patient candidates for a gene therapy trial.Glycosaminoglycan storage disorders: a review AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta)A review of gene therapy in canine and feline models of lysosomal storage disorders.Whole genome sequencing in cats, identifies new models for blindness in AIPL1 and somite segmentation in HES7.Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector.Gene therapy approaches for lysosomal storage disease: next-generation treatment.Gene therapy for mucopolysaccharidosis type VI is effective in cats without pre-existing immunity to AAV8.Pharmacological read-through of nonsense ARSB mutations as a potential therapeutic approach for mucopolysaccharidosis VI Genetically reprogrammed, liver-derived insulin-producing cells are glucose-responsive, but susceptible to autoimmune destruction in settings of murine model of type 1 diabetes.Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease.Gene Therapy in Tyrosinemia: Potential and Pitfalls.Improving lipoprotein profiles by liver-directed gene transfer of low density lipoprotein receptor gene in hypercholesterolaemia mice.Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI.Gene therapy for Mucopolysaccharidoses.

P2860

Q24622009-A70E082B-4ECB-47B6-A07C-5E88E8E61C8A Q26852308-359A22EE-1C9A-43EA-905E-0FE705BCDC68 Q27307010-B98A755E-F6FF-44B7-A6AF-CB3ADA9FDBF0 Q30513972-606E8CB7-B5E3-46FD-A109-9DFF0741B6F4 Q30563980-A557B598-8086-40B8-8414-3AFDC6D3DDF3 Q30583745-38CB950E-E7BA-4A62-B3A4-5092E8DA8DC3 Q34055904-5A29451B-CAEC-4800-980E-673332D6E5F8 Q34545883-C7AA0EB1-9D0F-402D-96BE-474E78165E10 Q35153234-E2AE6EAB-A24F-432D-A40F-52376416E846 Q35199296-9DD19373-0C25-4EB2-81DC-5850653E2271 Q35367599-A519BDF0-A6BF-475A-AAD6-B82072BCBA2F Q35568520-E1C19E4A-356F-41FF-ADF2-6D4D3A394536 Q35894676-05E4C47F-94C5-4718-A787-EC4D7608FC6E Q35974724-A1417377-AA94-4C8D-9B77-66FD219A2318 Q35988663-40462740-6EEE-40F9-9103-E53EE513F588 Q36147475-D2D7186D-79FF-4DB7-966D-AC15629B0A6B Q36634890-7B91EEE0-A1E1-4E72-A9D6-A51C7AC2B6A8 Q36662823-64FF8CB6-90B1-44A2-B063-88A9228FC596 Q36729250-3B4C0E53-7835-4B8C-A529-14118697C1AF Q37507816-C4D5DE86-BF3B-4850-AA4E-33BCFE55FD50 Q40103301-6F0D55AA-2D42-4694-AB57-9FF47B6FD193 Q40678976-733E27B3-833A-4631-9265-6D4BF227A191 Q41347222-603C4A99-5991-4F22-A4B7-0AA5E87EB1AC Q47558790-084A1EC3-F6EF-4F2C-9AAF-FD89EE5F4F2E

P2860

Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer

http://www.wikidata.org/entity/Q30498316

description

2010 nî lūn-bûn

@nan

2010 թուականի Նոյեմբերին հրատարակուած գիտական յօդուած

@hyw

2010 թվականի նոյեմբերին հրատարակված գիտական հոդված

@hy

2010年の論文

@ja

2010年論文

@yue

2010年論文

@zh-hant

2010年論文

@zh-hk

2010年論文

@zh-mo

2010年論文

@zh-tw

2010年论文

@wuu

name

Long-term amelioration of feli ...... V-mediated liver gene transfer

@ast

Long-term amelioration of feli ...... V-mediated liver gene transfer

@en

type

label

Long-term amelioration of feli ...... V-mediated liver gene transfer

@ast

Long-term amelioration of feli ...... V-mediated liver gene transfer

@en

prefLabel

Long-term amelioration of feli ...... V-mediated liver gene transfer

@ast

Long-term amelioration of feli ...... V-mediated liver gene transfer

@en

P1433

Q30498316-7C3A2C34-C823-464D-BCB9-78965A0065E6

P1476

Q30498316-4AE41A0C-679B-4CDF-9F4C-2718F13541FC

P2093

Q30498316-09EB5FE5-F29F-482F-89B8-DC4177BE7188

Q30498316-1506ABA8-BDC9-438B-9769-A62CF6F723A7

Q30498316-222373D6-6D8F-45AD-AD79-493E1D724B79

Q30498316-22D63635-CD3C-4555-AD4A-807AB24B9D0B

Q30498316-2B4178ED-E367-448C-AE57-AC1BDAC4B60C

Q30498316-400EB1CC-D569-4840-9C7D-6BE7CC5252AF

Q30498316-5648852B-665D-49FD-9A5B-3FAF37DB94EA

Q30498316-65323704-DA8B-4146-AF23-454E737B9A93

Q30498316-6B4A2359-671A-44E7-9792-17724E88700A

Q30498316-82480A0C-E584-4F89-966D-6B878A37E29E

P2860

Q30498316-0084C3B0-64F1-4091-A1F0-E92FE79D2AE5

Q30498316-12407336-4363-4CAA-97FF-9C6FE089FC43

Q30498316-13DA5176-3FFB-4A86-A9F2-860D5E88D3C2

Q30498316-1AC9D670-2BD8-4E68-82C9-AEB329F7D8D0

Q30498316-20E69019-F9D0-4B1B-8112-74EEF541AF4E

Q30498316-26EC95AC-0EA6-4183-94AF-E0B99C1EB745

Q30498316-378FEC4E-20BC-45CA-B0DE-7892EFFC2C4F

Q30498316-38FE9B73-8B3A-4FE3-85FC-56B010B88657

Q30498316-3D8D1F76-10A8-44BF-BB75-35950C11DF77

Q30498316-47FED1A7-FBCA-462D-B568-1262F8BCD957

P304

Q30498316-E82144AF-6CEF-4F71-BD0A-6F0CC3983577

P31

Q30498316-695F591F-57F7-4451-89DE-28C38EFC4814

P356

Q30498316-287A5882-7662-4F6C-9386-EE6C124E5B85

P433

Q30498316-40021221-59EC-49AD-B176-41F537824404

P478

Q30498316-52052036-7DBF-472E-BA68-518072C70C00

P50

Q30498316-1DB15449-7B22-4EF1-BB6C-E6E115C7B8F9

P577

Q30498316-26A40921-A61B-47F3-A882-0251E2241F85

P5875

Q30498316-FBBF2573-E1A8-4107-B9AC-957EF8529BDC

P698

Q30498316-ED5C6AFF-8E1F-4E71-8B82-FAEB20D664D6

P932

Q30498316-7B9958BD-E5D1-4DE2-BBD5-0A2D7FF2DD6D

P356

P1433

Molecular Therapy

P1476

Long-term amelioration of feli ...... V-mediated liver gene transfer

@en

P2093

Alessandra Tessitore

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

Anita Capalbo

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

Armida Faella

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

Elvira De Leonibus

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

Fabio Russo

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

Gabriella Cotugno

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

John Hopwood

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

Leah Levanduski

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

Mark Haskins

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

Meg M Sleeper

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

P2860

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

The multiple sulfatase deficiency gene encodes an essential and limiting factor for the activity of sulfatases

Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column.

Radiographic evaluation of bones and joints in mucopolysaccharidosis I and VII dogs after neonatal gene therapy.

The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques

Arylsulfatase B-deficient mucopolysaccharidosis in rats.

Mucopolysaccharidosis type VI: Structural and clinical implications of mutations in N-acetylgalactosamine-4-sulfatase.

Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer.

Enzyme replacement and enhancement therapies: lessons from lysosomal disorders.

Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates

P304

461-469

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

P31

scholarly article

P356

10.1038/MT.2010.257

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

P433

3

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

P478

19

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

P50

Alberto Auricchio

P577

2010-11-30T00:00:00Z

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

P5875

49647033

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

P698

21119624

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

P932

3048181

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