Gene therapy
US-10391201-B2 · Aug 27, 2019 · US
Method for improving retroviral transduction and gene editing in hematopoietic stem cells using clyclosporin H and UM171
US12485187B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-12485187-B2 |
| Application number | US-202418597488-A |
| Country | US |
| Kind code | B2 |
| Filing date | Mar 6, 2024 |
| Priority date | Apr 21, 2017 |
| Publication date | Dec 2, 2025 |
| Grant date | Dec 2, 2025 |
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.
Use of cyclosporin H (CsH) or a derivative thereof for increasing the efficiency of transduction of an isolated population of cells by a viral vector and/or increasing the efficiency of gene editing of an isolated population of cells when transduced by a viral vector.
First claim
Opening claim text (preview).
The invention claimed is: 1 . A method of transducing a population of CD34+ HSPCs comprising the steps of: (a) culturing the population of HSPCs in the presence of UM171; (b) contacting the population of HSPCs with cyclosporin H (CsH); and (c) transducing the population of HSPCs with a viral vector, wherein the viral vector is a lentiviral or gammaretroviral vector, and wherein the viral vector is pseudotyped to enter cells via an endocytosis-dependent mechanism. 2 . The method of claim 1 , wherein the viral vector is a VSV-g pseudotyped vector. 3 . The method of claim 1 , wherein the viral vector is a lentiviral vector. 4 . The method of claim 1 , wherein the viral vector is an integration-defective lentiviral vector (IDLV). 5 . The method of claim 1 , wherein the HSPCs are stimulated HSPCs. 6 . The method of claim 1 , wherein the population of HSPCs is cultured in step (a) in the further presence of StemRegenin 1 (SR-1). 7 . The method of claim 1 , wherein the population of HSPCs is cultured in step (a) in the further presence of prostaglandin E2. 8 . The method of claim 7 , wherein the prostaglandin E2 is 16-16 dimethyl prostaglandin E2. 9 . The method of claim 1 , wherein the viral vector comprises a nucleotide of interest. 10 . The method of claim 9 , wherein the nucleotide of interest is a donor template. 11 . The method of claim 1 , wherein the method further comprises the step: (d) contacting the population of HSPCs with a CRISPR/Cas system. 12 . The method of claim 11 , wherein the CRISPR/Cas system comprises a ribonucleoprotein (RNP) comprising Cas protein and gRNA. 13 . The method of claim 11 , wherein step (d) comprises electroporating the population of HSPCs. 14 . The method of claim 1 , wherein efficiency of transduction of the population of HSPCs by the viral vector is increased and/or efficiency of gene editing of the population of HSPCs when transduced by the viral vector is increased. 15 . The method of claim 1 , wherein the percentage of cells transduced by the viral vector is increased and/or the vector copy number per cell is increased. 16 . The method of claim 1 , wherein the method is carried out in vitro or ex vivo. 17 . The method of claim 1 , wherein the CsH is at a concentration of about 1-50 μM. 18 . The method of claim 1 , comprising a further step of enriching the population for CD34+/CD38-cells. 19 . The method of claim 1 , wherein the method comprises the steps of: (a) culturing the population of HSPCs in the presence of UM171; (b) contacting the population of HSPCs with CsH; (c) transducing the population of HSPCs with an IDLV comprising a donor template; (d) contacting the population of HSPCs with a CRISPR/Cas system. 20 . The method of claim 19 , wherein the CRISPR/Cas system comprises a ribonucleoprotein (RNP) comprising Cas protein and gRNA. 21 . The method of claim 1 , wherein the HSPCs are from umbilical cord blood, mobilized peripheral blood, or bone marrow. 22 . The method of claim 1 , wherein the HSPCs are CD34+CD38-HSPCs.
Assignees
Inventors
Classifications
viral genome or elements thereof as genetic vector · CPC title
- C12N15/86Primary
Viral vectors · CPC title
Increasing the copy number of the vector · CPC title
Haematopoietic stem cells; Uncommitted or multipotent progenitors · CPC title
Cyclosporins; Related peptides · 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 US12485187B2 cover?
- Use of cyclosporin H (CsH) or a derivative thereof for increasing the efficiency of transduction of an isolated population of cells by a viral vector and/or increasing the efficiency of gene editing of an isolated population of cells when transduced by a viral vector.
- Who is the assignee on this patent?
- Ospedale San Raffaele Srl, Fond Telethon Ets, Fond Telethon
- What technology area does this patent fall under?
- Primary CPC classification C12N15/86. Mapped technology areas include Chemistry & Metallurgy.
- When was this patent published?
- Publication date Tue Dec 02 2025 00:00:00 GMT+0000 (Coordinated Universal Time) (B2). Legal status and post-grant events are not shown on this page.
- What related patents are in patentsdb?
- We list 2 related publications on this page (citations in our corpus or others sharing the same primary CPC).