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Last Updated: April 25, 2024

Claims for Patent: 9,212,209

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Summary for Patent: 9,212,209

Title:	Screening methods for spinal muscular atrophy
Abstract:	Disclosed herein are compositions and methods for treatment of spinal muscular atrophy (SMA). In certain embodiments, compounds are provided that increase full-length survival of motor neuron (SMN) protein production by an SMN2 gene.
Inventor(s):	Androphy; Elliot (Indianapolis, IN), Cuny; Gregory D. (Houston, TX), Cherry; Jonathan (Carmel, IN), Glicksman; Marcie A. (Winchester, MA)
Assignee:	Indiana University Research and Technology Corporation (Indianapolis, IN)
Application Number:	13/941,099
Patent Claims:	1. A vector comprising: a survival motor neuron (SMN) promoter selected from the group consisting of a survival motor neuron 1 (SMN1) promoter and a survival motor neuron 2 (SMN2) promoter; a transcription start site; exons 1-8 of the SMN gene; a truncated intron 6 of the SMN gene; and a reporter gene. 2. The vector of claim 1, wherein the reporter gene encodes a reporter protein selected from the group consisting of luciferase, green fluorescent protein, and red fluorescent protein. 3. The vector of claim 1, wherein the truncated intron 6 comprises a 2 kilobase deletion. 4. A host cell comprising the vector of claim 1. 5. The host cell of claim 4 selected from the group consisting of a C33 cell; a HEK-293 cell; a spinal muscular atrophy type 1 (GM3813) fibroblast; and a 3814 (GM3814) fibroblast. 6. A vector comprising: a survival motor neuron 1 (SMN1) promoter; a transcription start site; exons 1-8 of the SMN1 gene; a truncated intron 6 of the SMN1 gene; and a reporter gene. 7. The vector of claim 6, wherein the reporter gene encodes a reporter protein selected from the group consisting of luciferase, green fluorescent protein, and red fluorescent protein. 8. The vector of claim 6, wherein the truncated intron 6 comprises a 2 kilobase deletion. 9. A host cell comprising the vector of claim 6. 10. The host cell of claim 9 selected from the group consisting of a C33 cell; a HEK-293 cell; a spinal muscular atrophy type 1 (GM3813) fibroblast; and a 3814 (GM3814) fibroblast. 11. A vector comprising: a survival motor neuron 2 (SMN2) promoter; a transcription start site; exons 1-8 of the SMN2 gene; a truncated intron 6 of the SMN2 gene; and a reporter gene. 12. The vector of claim 11, wherein the reporter gene encodes a reporter protein selected from the group consisting of luciferase, green fluorescent protein, and red fluorescent protein. 13. The vector of claim 11, wherein the truncated intron 6 comprises a 2 kilobase deletion. 14. A host cell comprising the vector of claim 11. 15. The host cell of claim 14 selected from the group consisting of a C33 cell; a HEK-293 cell; a spinal muscular atrophy type 1 (GM3813) fibroblast; and a 3814 (GM3814) fibroblast.

Details for Patent 9,212,209

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Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Approval Date	Patent No.	Expiredate
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	For Injection	103132	06/04/1986	⤷ Try a Trial	2032-07-13
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	For Injection	103132		⤷ Try a Trial	2032-07-13
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	Injection	103132		⤷ Try a Trial	2032-07-13
>Applicant	>Tradename	>Biologic Ingredient	>Dosage Form	>BLA	>Approval Date	>Patent No.	>Expiredate

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