Methods and materials for treating prader-willi syndrome
AssigneeUniversity of Pittsburgh—Of the Commonwealth System of Higher Education
InventorRobert David Nicholls
Life of this patent
Estimating the term…
Abstract
This document provides methods and materials for treating a mammal (e.g., a human) having Prader-Willi syndrome (PWS). For example, adeno-associated virus (AAV) vectors can be designed to express one or more nucleic acids associated with PWS (PWS nucleic acids) and administered to a mammal (e.g., a human) having PWS to treat the mammal.
Claims32
An adeno-associated virus comprising a single-stranded DNA, wherein said single-stranded DNA comprises a first inverted terminal repeat (ITR) sequence followed by an engineered DNA sequence followed by a second ITR sequence, wherein said engineered DNA sequence comprises (a) a first DNA sequence comprising a first promotor sequence operably linked to first DNA segment comprising a nucleotide sequence encoding a necdin polypeptide and (b) a second DNA sequence comprising a second promotor sequence operably linked to a second DNA segment comprising a nucleotide sequence encoding a SNURF polypeptide, a nucleotide sequence encoding a SNRPN polypeptide, a nucleotide sequence encoding a SNORD107 small nucleolar RNA (snoRNA), a nucleotide sequence encoding a SNORD64 snoRNA, a nucleotide sequence encoding a SNORD116 snoRNA, a nucleotide sequence encoding a SNORD115 snoRNA, and a nucleotide sequence encoding a SNORD109 snoRNA, wherein each of said first promotor sequence and said second promotor sequence comprises at least four motifs having the DNA sequence set forth in SEQ ID NO:
- 10 (5′-YGCGCANGCGCR-3′), wherein 8 to 12 intervening nucleotides are located between each adjacent motif of said at least four motifs, and wherein delivery of said adeno-associated virus to a cell within said mammal results in expression of said necdin polypeptide, said SNURF polypeptide, said SNRPN polypeptide, said SNORD107 snoRNA, said SNORD64 snoRNA, said SNORD116 snoRNA, said SNORD115 snoRNA, and said SNORD109 snoRNA.
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A method for increasing expression of a necdin polypeptide, a SNURF polypeptide, a SNRPN polypeptide, a SNORD107 snoRNA, a SNORD64 snoRNA, a SNORD116 snoRNA, a SNORD115 snoRNA, and a SNORD109 snoRNA by cells within a mammal, wherein said method comprises administering an adeno-associated virus to said mammal, thereby increasing expression of said necdin polypeptide, said SNURF polypeptide, said SNRPN polypeptide, said SNORD107 snoRNA, said SNORD64 snoRNA, said SNORD116 snoRNA, said SNORD115 snoRNA, and said SNORD109 snoRNA by said cells within said mammal,
- wherein said adeno-associated virus comprises a single-stranded DNA, wherein said single-stranded DNA comprises a first inverted terminal repeat (ITR) sequence followed by an engineered DNA sequence followed by a second ITR sequence, wherein said engineered DNA sequence comprises (a) a first DNA sequence comprising a first promotor sequence operably linked to first DNA segment comprising a nucleotide sequence encoding said necdin polypeptide and (b) a second DNA sequence comprising a second promotor sequence operably linked to a second DNA segment comprising a nucleotide sequence encoding said SNURF polypeptide, a nucleotide sequence encoding said SNRPN polypeptide, a nucleotide sequence encoding said SNORD107 snoRNA, a nucleotide sequence encoding said SNORD64 snoRNA, a nucleotide sequence encoding said SNORD116 snoRNA, a nucleotide sequence encoding said SNORD115 snoRNA, and a nucleotide sequence encoding said SNORD109 snoRNA, wherein each of said first promotor sequence and said second promotor sequence comprises at least four motifs having the DNA sequence set forth in SEQ ID NO:10 (5′-YGCGCANGCGCR-3′), wherein 8 to 12 intervening nucleotides are located between each adjacent motif of said at least four motifs.
A method for treating Prader-Willi syndrome, wherein said method comprises administering an adeno-associated virus of to a mammal having Prader-Willi syndrome, wherein expression of a necdin polypeptide, a SNURF polypeptide, a SNRPN polypeptide, a SNORD107 snoRNA, a SNORD64 snoRNA, a SNORD116 snoRNA, a SNORD115 snoRNA, and a SNORD109 snoRNA by cells within said mammal is increased following said administering step, and wherein severity of a symptom of said Prader-Willi syndrome is reduced following said administering step
- wherein said adeno-associated virus comprises a single-stranded DNA, wherein said single-stranded DNA comprises a first inverted terminal repeat (ITR) sequence followed by an engineered DNA sequence followed by a second ITR sequence, wherein said engineered DNA sequence comprises (a) a first DNA sequence comprising a first promotor sequence operably linked to first DNA segment comprising a nucleotide sequence encoding said necdin polypeptide and (b) a second DNA sequence comprising a second promotor sequence operably linked to a second DNA segment comprising a nucleotide sequence encoding said SNURF polypeptide, a nucleotide sequence encoding said SNRPN polypeptide, a nucleotide sequence encoding said SNORD107 snoRNA, a nucleotide sequence encoding said SNORD64 snoRNA, a nucleotide sequence encoding said SNORD116 snoRNA, a nucleotide sequence encoding said SNORD115 snoRNA, and a nucleotide sequence encoding said SNORD109 snoRNA, wherein each of said first promotor sequence and said second promotor sequence comprises at least four motifs having the DNA sequence set forth in SEQ ID NO:
- 10 (5′-YGCGCANGCGCR-3′), wherein 8 to 12 intervening nucleotides are located between each adjacent motif of said at least four motifs.
Dependencies are inferred from the claim text.