Rna replicon for improving gene expression and use thereof
US-2024417751-A1 · Dec 19, 2024 · US
Excision of retroviral nucleic acid sequences
US2020392487A1 · US · A1
| Field | Value |
|---|---|
| Publication number | US-2020392487-A1 |
| Application number | US-202016926427-A |
| Country | US |
| Kind code | A1 |
| Filing date | Jul 10, 2020 |
| Priority date | Feb 15, 2016 |
| Publication date | Dec 17, 2020 |
| Grant date | — |
How to read this patent
A practical reading order for non-experts. Skip the full description unless you need deep technical detail.
- Title
What the patent document calls the invention.
- Abstract
A short plain-language summary of the technical disclosure.
- Assignees and inventors
Who owns or filed the patent and who is credited as inventor.
- Key dates
Filing, priority, publication, and grant dates set the timeline.
- First independent claim
The legal scope of protection — read this for what is actually claimed.
- CPC / IPC classifications
Technology tags used to group this patent with similar filings.
- Citations and related patents
Prior art links and similar publications in this corpus.
Abstract
Official abstract text for this publication.
Compositions for the in vivo delivery of a gene editing CRISPR/Cas9 complex was developed to eliminate integrated retroviral DNA sequences from latently infected human cells and animal disease models
First claim
Opening claim text (preview).
1 .- 46 . (canceled) 47 . An adeno-associated viral vector serotype 9 (AAV9) expression vector, comprising a nucleic acid sequence encoding: (a) a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease; (b) a first guide RNA (gRNA), the first gRNA being complementary to a target sequence within a long terminal repeat (LTR) of a human immunodeficiency virus (HIV) sequence, and wherein the first gRNA comprises a sequence having at least 80% sequence identity to SEQ ID NO: 207; and (c) a second gRNA, the second gRNA being complementary to a target sequence within a GagD region of the HIV sequence. 48 . The AAV9 expression vector of claim 47 , wherein the CRISPR-associated endonuclease is Cas9 or a homologue thereof. 49 . The AAV9 expression vector of claim 47 , wherein the first gRNA comprises a sequence having at least 90% sequence identity to SEQ ID NO: 207. 50 . The AAV9 expression vector of claim 47 , wherein the first gRNA comprises a sequence according to SEQ ID NO: 207. 51 . An adeno-associated viral vector serotype 9 (AAV9) expression vector, comprising a nucleic acid sequence encoding: (a) a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease; (b) a first guide RNA (gRNA), the first gRNA being complementary to a target sequence within a long terminal repeat (LTR) of a human immunodeficiency virus (HIV) sequence; and (c) a second gRNA, the second gRNA being complementary to a target sequence within a GagD region of the HIV sequence, and wherein the second gRNA comprises a sequence having at least 80% sequence identity to SEQ ID NO: 92. 52 . The AAV9 expression vector of claim 51 , wherein the CRISPR-associated endonuclease is Cas9 or a homologue thereof. 53 . The AAV9 expression vector of claim 51 , wherein the second gRNA comprises a sequence having at least 90% sequence identity to SEQ ID NO: 92. 54 . The AAV9 expression vector of claim 51 , wherein the second gRNA comprises a sequence according to SEQ ID NO: 92. 55 . An adeno-associated viral vector serotype 9 (AAV9) expression vector, comprising a nucleic acid sequence encoding: (a) a Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated endonuclease; (b) a first guide RNA (gRNA), the first gRNA being complementary to a target sequence within a long terminal repeat (LTR) of a human immunodeficiency virus (HIV) sequence, and wherein the first gRNA comprises a sequence having at least 80% sequence identity to SEQ ID NO: 207; and (c) a second gRNA, the second gRNA being complementary to a target sequence within a GagD region of the HIV sequence, and wherein the second gRNA comprises a sequence having at least 80% sequence identity SEQ ID NO: 92. 56 . The AAV9 expression vector of claim 55 , wherein the CRISPR-associated endonuclease is Cas9 or a homologue thereof. 57 . The AAV9 expression vector of claim 55 , wherein the first gRNA comprises a sequence having at least 90% sequence identity to SEQ ID NO: 207. 58 . The AAV9 expression vector of claim 55 , wherein the first gRNA comprises a sequence according to SEQ ID NO: 207. 59 . The AAV9 expression vector of claim 55 , wherein the second gRNA comprises a sequence having at least 90% sequence identity to SEQ ID NO: 92. 60 . The AAV9 expression vector of claim 55 , wherein the second gRNA comprises a sequence according to SEQ ID NO: 92.
Assignees
Inventors
Classifications
- A61K48/00Primary
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy · CPC title
containing a nuclear localisation signal · CPC title
against retroviridae, e.g. HIV · CPC title
Animal models for infectious diseases · CPC title
Murine · CPC title
Patent family
Related publications grouped by family.
External sources
Frequently asked questions
Answers are generated from the same data shown on this page.
- What does patent US2020392487A1 cover?
- Compositions for the in vivo delivery of a gene editing CRISPR/Cas9 complex was developed to eliminate integrated retroviral DNA sequences from latently infected human cells and animal disease models
- Who is the assignee on this patent?
- Univ Temple
- What technology area does this patent fall under?
- Primary CPC classification A61K48/00. Mapped technology areas include Human Necessities.
- When was this patent published?
- Publication date Thu Dec 17 2020 00:00:00 GMT+0000 (Coordinated Universal Time) (A1). Legal status and post-grant events are not shown on this page.
- What related patents are in patentsdb?
- We list 8 related publications on this page (citations in our corpus or others sharing the same primary CPC).