Gene therapy for lysosomal storage diseases (LSDs) in large animal models.
about
Gene therapy in large animal models of human genetic diseases. IntroductionTherapeutic approaches for lysosomal storage diseasesA digital atlas of the dog brainProtein replacement therapy and gene transfer in canine models of hemophilia A, hemophilia B, von willebrand disease, and factor VII deficiency.Lysosomal storage disease: gene therapy on both sides of the blood-brain barrier.Lentiviral-mediated gene transfer to the sheep brain: implications for gene therapy in Batten disease.Evaluation of adeno-associated viral vectors for liver-directed gene transfer in dogs.Therapeutic approaches to the challenge of neuronal ceroid lipofuscinoses.Adeno-associated virus serotypes 9 and rh10 mediate strong neuronal transduction of the dog brain.Disease models for the development of therapies for lysosomal storage diseases.Strategies for delivery of therapeutics into the central nervous system for treatment of lysosomal storage disorders.New biotechnological and nanomedicine strategies for treatment of lysosomal storage disordersViral vector-mediated gene transfer for CNS disease.Therapeutic approaches for neuronopathic lysosomal storage disorders.Lessons learnt from animal models: pathophysiology of neuropathic lysosomal storage disorders.Pathology and current treatment of neurodegenerative sphingolipidoses.Newborn screening for lysosomal storage disorders.Emerging therapies for neurodegenerative lysosomal storage disorders - from concept to reality.Gene therapy approaches for lysosomal storage disorders, a good model for the treatment of mendelian diseases.Treatment options for lysosomal storage disorders: developing insights.Animal models for metabolic, neuromuscular and ophthalmological rare diseases.Presumptive Spontaneous Lysosomal Storage-Like Disease in a Crl:CD1(ICR) Mouse.Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats.
P2860
Q33808511-064BFAA4-FC85-4D62-8B43-9E087FF32118Q34311420-063F5361-15C7-4B69-BDF7-DE13C23A4756Q34533109-E181F047-5EA1-4846-BF88-885B27023933Q35006603-1272EFD5-FFE4-4C10-972F-64C64A64DC29Q35034401-1A0CF4B3-F264-4AC0-9FAA-9CD8300AC6E7Q35174910-93DF4D57-DF76-4E37-938B-BECA040EC427Q35174932-F05259A6-46F0-42AE-AAE9-75D4B907A772Q36784566-14F0D100-EE51-4428-B2D6-F2E1439B5A58Q37434391-5A33CB46-22C5-496A-8A1B-76911B71E9B0Q37619231-6B342658-797D-455F-9E12-3791CF8F100EQ37672410-2BF15FE5-0065-49B7-BBB8-B9A1AE2D5BE2Q37683644-2D49697A-F5F5-4AE2-BBBB-43734F6E2807Q37687420-9BA9A03E-11D8-4319-A693-B883A818C56FQ37693817-17A1F9DD-F850-4042-B8CC-001D5ABF8A34Q37744753-428AB11A-DA8E-4FE3-97CD-94C32DE5C6C5Q37781746-666FFA3E-6C9E-4EA2-A174-481869AE2A00Q37839905-612A0EF9-1BDF-4C4E-BD2A-68E67926F10DQ37876354-1D645C89-20BF-40B3-B3D1-ACC550A37471Q37994717-C14E5FE1-0EFB-4A14-89E1-230A3D11D839Q38046227-5A2B167F-B1B8-4873-9655-DF87AC5F3F8EQ38089518-443DD60E-836C-4D00-BB7C-EFD3E1399072Q40323432-614B7213-5A40-4FDB-859D-CFB2E188D6CBQ45872026-2E86B913-46D9-48C8-BBA5-7AD639C46279
P2860
Gene therapy for lysosomal storage diseases (LSDs) in large animal models.
description
2009 nî lūn-bûn
@nan
2009年の論文
@ja
2009年論文
@yue
2009年論文
@zh-hant
2009年論文
@zh-hk
2009年論文
@zh-mo
2009年論文
@zh-tw
2009年论文
@wuu
2009年论文
@zh
2009年论文
@zh-cn
name
Gene therapy for lysosomal storage diseases (LSDs) in large animal models.
@ast
Gene therapy for lysosomal storage diseases (LSDs) in large animal models.
@en
type
label
Gene therapy for lysosomal storage diseases (LSDs) in large animal models.
@ast
Gene therapy for lysosomal storage diseases (LSDs) in large animal models.
@en
prefLabel
Gene therapy for lysosomal storage diseases (LSDs) in large animal models.
@ast
Gene therapy for lysosomal storage diseases (LSDs) in large animal models.
@en
P2860
P356
P1433
P1476
Gene therapy for lysosomal storage diseases (LSDs) in large animal models.
@en
P2093
Mark Haskins
P2860
P304
P356
10.1093/ILAR.50.2.112
P577
2009-01-01T00:00:00Z