Biomimetic CRISPR Nanoformulations for Targeted Gene Editing in Thallasemia and Sickle Cell Disease
About This Grant
PROJECT SUMMARY Hematopoietic stem cells (HSCs) are essential for the continuous production and regeneration of the hematopoietic system. Among them, long-term hematopoietic stem cells (LT-HSCs) possess the unique ability to self-renew and differentiate into all blood cell types, making them critical for maintaining blood homeostasis. Leveraging the therapeutic potential of LT-HSCs, especially in the field of gene editing, holds great promise for addressing genetic defects associated with various hematological disorders. Gene editing technologies, such as CRISPR, have revolutionized molecular biology by enabling precise modifications of cell genetic material. In the context of LT-HSCs, gene editing offers a potential solution for a wide range of hematological disorders, including inherited blood diseases and malignancies. A significant application of gene editing in HSCs involves inducing fetal hemoglobin (FH) production as a substitute for adult hemoglobin. Genetic mutations impacting the BCL11A binding site on the γ-globin gene have demonstrated therapeutic potential for beta-hemoglobinopathies. Currently, achieving FH induction in HSCs requires isolating CD34+ cells and applying CRISPR components ex vivo through electroporation. However, this process is time-consuming, lacks specificity for LT-HSCs, and is limited to well-equipped facilities, making it less accessible, particularly in resource-limited regions where most cases of beta hemoglobinopathies occur. Additionally, patient HSCs may not be suitable for ex vivo treatment. Thus, there is a critical need to develop in vivo CRISPR delivery systems to make this technology widely available. Indeed, our preliminary data show that exosomes have highly specific interactions with long-term hematopoietic stem cells (LT-HSCs). Using a bottom-up nanotechnology approach we have developed biomimetic LNPs that can encapsulate CRISPR in its highly effective RNP form. The proposed research is innovative because it aims to investigate the potential of biomimetic CRISPR delivery vehicles, inspired by exosomes that specifically interact with LT-HSCs, as gene editing delivery systems to induce FH production. By incorporating highly specific exosome components, our Exo-CRISPR nanoformulations, can be engineered to selectively target LT-HSCs. This approach offers the advantage of enhancing the efficiency and specificity of CRISPR-based gene editing in LT-HSCs, maximizing therapeutic impact. Overall, this project aims to develop a targeted and efficient gene editing delivery system using Exo-CRISPR LNPs to induce FH production, offering a promising therapeutic strategy for beta hemoglobinopathies. The project's findings will contribute to advancing the field of gene editing and improving the treatment outcomes for patients with hematopoietic disorders.
Grant Summary
Biomimetic CRISPR Nanoformulations for Targeted Gene Editing in Thallasemia and Sickle Cell Disease is a NHLBI - National Heart Lung and Blood Institute grant providing up to $792K for university, nonprofit, healthcare org. Applications are due 2031-04-30 (open). Check eligibility and apply with FindGrants.
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Eligibility
How to Apply
Up to $792K
2031-04-30
- 1Confirm your organization is eligible for Biomimetic CRISPR Nanoformulations for Targeted Gene Editing in Thallasemia and Sickle Cell Disease from NHLBI - National Heart Lung and Blood Institute, checking organization type, location, and any population or project requirements.
- 2Gather the required documents and information, including your organization details, project plan, and budget figures.
- 3Draft your application narrative and budget addressing the funder's priorities and review criteria. FindGrants can draft each section for you to review and edit.
- 4Review every section against the requirements checklist, then export a submission-ready application pack and submit it to NHLBI - National Heart Lung and Blood Institute before the deadline.
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Biomimetic CRISPR Nanoformulations for Targeted Gene Editing in Thallasemia and Sickle Cell Disease: Frequently Asked Questions
Who is eligible for the Biomimetic CRISPR Nanoformulations for Targeted Gene Editing in Thallasemia and Sickle Cell Disease?
Biomimetic CRISPR Nanoformulations for Targeted Gene Editing in Thallasemia and Sickle Cell Disease is offered by NHLBI - National Heart Lung and Blood Institute and is generally open to university, nonprofit, healthcare org. It is open to organizations nationwide unless the funder specifies otherwise. Review the specific eligibility terms before applying, since funders set their own requirements around organization type, location, and the population or project being served.
How much funding does the Biomimetic CRISPR Nanoformulations for Targeted Gene Editing in Thallasemia and Sickle Cell Disease provide?
Biomimetic CRISPR Nanoformulations for Targeted Gene Editing in Thallasemia and Sickle Cell Disease provides up to $792K per award from NHLBI - National Heart Lung and Blood Institute. Actual award sizes depend on the scope of your project, available program funds, and the number of applicants, so build a budget that reflects realistic, allowable costs rather than the maximum figure.
When is the Biomimetic CRISPR Nanoformulations for Targeted Gene Editing in Thallasemia and Sickle Cell Disease deadline?
Applications for Biomimetic CRISPR Nanoformulations for Targeted Gene Editing in Thallasemia and Sickle Cell Disease are due 2031-04-30 (open). Because deadlines can change, verify the date with the funder, NHLBI - National Heart Lung and Blood Institute, and give yourself enough time to prepare a complete, competitive application before the close date.
How do you apply for the Biomimetic CRISPR Nanoformulations for Targeted Gene Editing in Thallasemia and Sickle Cell Disease?
To apply for Biomimetic CRISPR Nanoformulations for Targeted Gene Editing in Thallasemia and Sickle Cell Disease, confirm your eligibility, gather the required documents, and prepare a narrative and budget that address the funder's priorities. FindGrants guides you step by step and can draft each section, then exports a submission-ready application pack for this grant from NHLBI - National Heart Lung and Blood Institute.