Gene therapy for alzheimer`s disease
AssigneesThe Brigham and Women's Hospital, Inc., The General Hospital Corporation
InventorsJie Shen, Raymond J. Kelleher, III
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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).
Claims54
A method of treating a neurodegenerative disease, disorder, or condition, the method comprising administering to a subject in need of treatment a polynucleotide encoding a presenilin 1 (PS1) and/or presenilin 2 (PS2) protein, wherein the subject has one or more mutations in at least one allele of PSEN1 and/or PSEN2 that encodes a dominant negative PS1 or PS2 protein isoform.
The method of any one of the preceding claims, comprising administering the polynucleotide in a vector, exosome, or lipid-based nanoparticle (LNP).
The method of any one of the preceding claims, wherein the polynucleotide comprises a presenilin 1 (PSEN1) gene or mRNA.
The method of any one of the preceding claims, wherein the polynucleotide comprises a presenilin 2 (PSEN2) gene or mRNA.
The method of any one of the preceding claims, wherein the polynucleotide comprises a presenilin 1 (PSEN1) gene or mRNA and a presenilin 2 (PSEN2) gene or mRNA.
The method of any one of the preceding claims, wherein the polynucleotide encoding PS1 comprises a nucleotide sequence at least 60%, 65%, 70%, 75%, 80%, 85%, 90%, 95%, 96%, 97%, 98%, 99% identical to SEQ ID NO:1 or SEQ ID NO:2.
The method of any one of the preceding claims, wherein the polynucleotide encoding PS2 comprises a nucleotide sequence at least 60%, 65%, 70%, 75%, 80%, 85%, 90%, 95%, 96%, 97%, 98%, 99% identical to SEQ ID NO:3 or SEQ ID NO:4.
The method of any one of the preceding claims, wherein the polynucleotide encoding a presenilin 1 (PS1) and/or presenilin 2 (PS2) is operably linked to a promoter.
The method of any one of the preceding claims, wherein the polynucleotide encoding PS1 and/or PS2 is administered to the CNS of the subject in need of treatment.
A polynucleotide sequence encoding a presenilin 1 (PS1) and/or presenilin 2 (PS2) protein, for use in treating a neurodegenerative disease, disorder or condition in a subject, wherein the subject has one or more mutations in at least one allele of PSEN1 and/or PSEN2 that encodes a dominant negative PS1 or PS2 protein isoform.
A vector comprising a polynucleotide sequence encoding a presenilin 1 (PS1) and/or presenilin 2 (PS2) protein, wherein the PS1 and/or PS2 encoding sequence is operably linked to a promotor that drives expression of the PS1 and/or PS2 in the brain, for use in treating a neurodegenerative disease, disorder or condition in a subject, wherein the subject has one or more mutations in at least one allele of PSEN1 and/or PSEN2 that encodes a dominant negative PS1 or PS2 protein isoform.
Dependencies are inferred from the claim text.