Orphan designation: Adenovirus associated viral vector serotype 5 containing the human RPE65 gene, Treatment of leber's congenital amaurosis

Case Medical Research ◽

10.31525/cmr-12e1a94 ◽

2019 ◽

Author(s):

Keyword(s):

Viral Vector ◽

Orphan Designation ◽

Gene Treatment ◽

Leber’S Congenital Amaurosis

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Orphan designation: Recombinant adeno-associated viral vector serotype 5 encoding Staphylococcus aureus Cas9 endonuclease and two guide RNAs complementary to two regions of intron 26 of the CEP290 gene, Treatment of leber's congenital amaurosis

Case Medical Research ◽

10.31525/cmr-178003a ◽

2019 ◽

Author(s):

Keyword(s):

Staphylococcus Aureus ◽

Viral Vector ◽

Orphan Designation ◽

Gene Treatment ◽

Leber’S Congenital Amaurosis

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Orphan designation: Adenovirus-associated viral vector serotype 5 containing the human RPGR gene, Treatment of retinitis pigmentosa

Case Medical Research ◽

10.31525/cmr-12e1a9d ◽

2019 ◽

Author(s):

Keyword(s):

Retinitis Pigmentosa ◽

Viral Vector ◽

Orphan Designation ◽

Gene Treatment ◽

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Orphan designation: Adenovirus associated viral vector serotype 8 containing the human AIPL1 gene, Treatment of leber's congenital amaurosis

Case Medical Research ◽

10.31525/cmr-12e1a9a ◽

2019 ◽

Author(s):

Keyword(s):

Viral Vector ◽

Orphan Designation ◽

Gene Treatment ◽

Leber’S Congenital Amaurosis

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Orphan designation: Adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene, Treatment of haemophilia A

Case Medical Research ◽

10.31525/cmr-3996ed ◽

2018 ◽

Author(s):

Keyword(s):

Factor Viii ◽

Viral Vector ◽

Coagulation Factor ◽

Coagulation Factor Viii ◽

Haemophilia A ◽

Factor Viii Gene ◽

Orphan Designation ◽

Gene Treatment ◽

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Orphan designation: Adeno-associated viral vector serotype 2 containing the human REP1 gene, Treatment of choroideraemia

Case Medical Research ◽

10.31525/cmr-399699 ◽

2019 ◽

Author(s):

Keyword(s):

Viral Vector ◽

Orphan Designation ◽

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Orphan designation: Adeno-associated viral vector serotype 9 containing the human SMN gene, Treatment of spinal muscular atrophy

Case Medical Research ◽

10.31525/cmr-1e4edc3 ◽

2019 ◽

Author(s):

Keyword(s):

Spinal Muscular Atrophy ◽

Viral Vector ◽

Muscular Atrophy ◽

Orphan Designation ◽

Gene Treatment ◽

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Orphan designation: Adeno-associated viral vector serotype 8 containing the human alpha-galactosidase A gene, Treatment of Fabry disease

Case Medical Research ◽

10.31525/cmr-128cc2f ◽

2019 ◽

Author(s):

Keyword(s):

Fabry Disease ◽

Viral Vector ◽

Orphan Designation ◽

Gene Treatment ◽

Alpha Galactosidase

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Orphan designation: Adeno-associated viral vector serotype 8 containing the human MTM1 gene, Treatment of X-linked myotubular myopathy

Case Medical Research ◽

10.31525/cmr-10e0aa5 ◽

2019 ◽

Author(s):

Keyword(s):

Viral Vector ◽

Myotubular Myopathy ◽

Orphan Designation ◽

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Orphan designation: adeno-associated viral vector serotype hu68 containing the human SMN1 gene, Treatment of spinal muscular atrophy

Case Medical Research ◽

10.31525/cmr-6939f8 ◽

2018 ◽

Author(s):

Keyword(s):

Spinal Muscular Atrophy ◽

Viral Vector ◽

Muscular Atrophy ◽

Smn1 Gene ◽

Orphan Designation ◽

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Orphan designation: Adeno-associated viral vector serotype 2 containing the human REP1 gene, Treatment of choroideraemia

Case Medical Research ◽

10.31525/cmr-ff5e16 ◽

2019 ◽

Author(s):

Keyword(s):

Viral Vector ◽

Orphan Designation ◽

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