Vector system for expressing regulatory RNA
AssigneeAmerican Gene Technologies International Inc.
InventorsTyler Lahusen, Lingzhi Xiao, Charles David Pauza
Life of this patent
Estimating the term…
Abstract
Viral vectors, lentiviral particles, and modified cells are disclosed. They encode or express a small RNA capable of targeting the KIF11 gene. In embodiments, the viral vectors and lentiviral particles further comprise and a KIF11 gene whose non-coding region has been modified such that it is resistant to activity by the small RNA.
Claims17
A modified mesenchymal stem cell comprising a mesenchymal stem cell infected with a lentiviral particle, wherein the lentiviral particle comprises:
- an envelope protein capable of infecting the mesenchymal stem cell;
- and
- a first nucleotide sequence encoding a small RNA capable of binding a non-coding region of a host copy of KIF11, wherein the first nucleotide sequence encoding a small RNA is present in the modified mesenchymal stem cell at between about 1 and about 10 copies per cell.
A method of producing a modified mesenchymal stem cell, the method comprising:
- infecting a mesenchymal stem cell with an effective amount of a lentiviral particle, wherein the lentiviral particle comprises:
- an envelope protein capable of infecting the mesenchymal stem cell;
- and
- a first nucleotide sequence encoding a small RNA capable of binding a non-coding region of a host copy of KIF11, wherein the first nucleotide sequence is present in the modified mesenchymal stem cell at between about 1 and about 10 copies per cell.
A lentiviral particle produced by a packaging cell and capable of infecting a target cell, the lentiviral particle comprising:
- an envelope protein capable of infecting the target cell;
- and
- a first nucleotide sequence encoding a small RNA capable of binding a non-coding region of a host copy of KIF11,
- wherein the non-coding region of the host copy of KIF11 is a 3′ untranslated region or a 5′ untranslated region,
- wherein the lentiviral particle further comprises a second nucleotide sequence encoding a KIF11 gene or a variant thereof, wherein the KIF11 gene or the variant thereof lacks at least one sequence portion,
- wherein the sequence portion is in a non-coding region of the KIF11 gene, and wherein the non-coding region of the KIF11 gene is in at least one of a 5′ untranslated region or a 3′ untranslated region, and
- wherein the target cell is a mesenchymal stem cell.
Dependencies are inferred from the claim text.