Granted Patent
B2
US 12,497,608 · App. 17/057,001 · Granted Dec 16, 2025
Gene therapy for Alzheimer's disease
C12N9/6478A61K9/0019A61K9/0085A61K9/5123A61P25/28C12N7/00C12N15/86C12Y304/23045C12Y304/23046A61K38/00A61K48/00C12N2710/16132C12N2710/16143
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Abstract
The present disclosure provides, among other things, methods for using presenilin based gene therapy to treat neurodegenerative dementia including, but not limited to Alzheimers disease, frontotemporal dementia, frontotemporal lobar degeneration, Picks disease, Lewy body dementia, memory loss, and cognitive impairment including mild cognitive impairment (MCI).
Claims (3)
1 . A method of treating one or more symptoms of familial Alzheimer's Disease in a human subject having human presenilin-1 (PSEN1) gene mutation L435F as compared to SEQ ID NO: 17, the method comprising administering by intracerebroventricular injection to the subject an AAV9 vector comprising a polynucleotide encoding the amino acid sequence of SEQ ID NO: 5 or SEQ ID NO: 6 operably linked to a synapsin I or an alpha-calcium/calmodulin kinase 2A promoter.
2 . The method of claim 1 , wherein the polynucleotide encoding the amino acid sequence is operably linked to a synapsin I promoter.
3 . The method of claim 1 , wherein the polynucleotide encoding the amino acid sequence is operably linked to alpha-calcium/calmodulin kinase 2A promoter.
Assignments (2)
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Mar 31, 2021
From: KELLEHER, RAYMOND J., III
To: THE GENERAL HOSPITAL CORPORATION
Reel/Frame 055777/0205 →
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Mar 31, 2021
From: SHEN, JIE
To: THE BRIGHAM AND WOMEN'S HOSPITAL, INC.
Reel/Frame 055777/0213 →
Continuity (2)
Provisional Application
62675003
· May 22, 2018
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