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Cystic Fibrosis Foundation (CFF) – Advancing Gene Editing Technologies and Tools for Cystic Fibrosis – Pilot and Feasibility Studies
Sponsor: Cystic Fibrosis Foundation (CFF) 		Closing Date: 12-Mar-2019
The Cystic Fibrosis Foundation (CFF) is pursuing genome editing as a therapeutic strategy to treat individuals with cystic fibrosis. To this end, the Cystic Fibrosis Foundation has announced an upcoming new Request For Applications for “Advancing Gene Editing Technologies and Tools for Cystic Fibrosis.”

CFF encourages investigators involved in gene editing to apply and requires no previous experience in cystic fibrosis research. A goal of the program will be to connect experts in gene editing with experts in cystic fibrosis to create a collaborative network of investigators who can synergize to move the editing field forward.

Key Dates
Full Applications due to UQ R&I: 26 February 2019
Full Applications due to CFF: 12 March 2019
Anticipated announcement: May 2019

Overview

• Aims
Proposals will focus on exploring technologies related to gene editing and generating tools for assessing editing and generating tools for assessing editing. These include, but are not limited to, studies aimed at:
• Creating tools, including animal models, cell lines or organoids, that will be generally applicable and facilitate or accelerate development and assessment of various gene editing strategies.
• Developing and optimizing novel gene editing technology platforms and strategies to target the CFTR gene locus.
• Improving our understanding of potential effects of CFTR gene editing on gene and chromatin topology as related to regulation of CFTR expression.
• Developing and applying assays to assess off-target effects and adverse events of CFTR gene editing in vitro and/or in vivo.
• Developing biological endpoints and assays for early in vivo efficacy signals of CFTR gene editing.
• Understanding and developing assays to monitor potential immune responses to gene editing. This may include responses to the delivery vehicle, cargo, or CFTR protein.
• Utilizing CF and/or non-CF animal models to address optimal delivery approaches, appropriate cell targets for long-term correction, dosing, safety, and phenotypic correction of CF pathology.
• Identifying the threshold of CFTR gene editing needed to reach therapeutic relevance.
• Identifying and overcoming barriers for delivery of gene editing cargo into cells relevant to CF.

• Eligibility
• Applicants must be a M.D., Ph.D. or dual M.D., Ph.D scientist.
• Candidates who are postdoctoral fellows should apply to the Advancing Gene Editing Technologies and Tools for Cystic Fibrosis Postdoctoral Fellowship program.

• Funding
The duration of the Research Grant is up to 2 years, with funding up to US $75,000 per year.

• Overheads
The maximum rate of overheads allowed by CFF for this scheme is 8%, which is in addition to the US $200,000 per year allowed for direct research costs.

How to Apply

Step 1. Completing the application
Log into proposalcentral.altum.com to access key documents and to complete the application.

Step 2. UQ R&I internal review and feedback
For UQ R&I internal review and feedback, email your application and completed Funding Application Coversheet to your UQ R&I Office of Sponsored Research RAO.
Website: http://www.cff.org/Research/

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