NIH-funded research NIH-funded research

New tools to precisely edit and read DNA and gene activity for treating disease

Official title: Center for Genomic Editing and Recording: Development and Application of Next-Generation Genome and Epigenome Editing Methods to Advance the Study and Treatment of Human Disease

Funded at Whitehead Institute for Biomedical Res · NIH-11123231 · Data updated 2026-11-12

Researchers are building next-generation tools to change and record DNA and gene activity to help people with genetic diseases and cancer get safer, more targeted care.

Quick facts

Grant typeNIH-funded research
Study typeNIH-funded research
Funding institutionWhitehead Institute for Biomedical Res
NIH-funded
Lab location1 site (Cambridge, United States)
Project startAugust 8, 2017
Project endMay 31, 2027
Funding year2025
Award (this year)$2,450,000
NIH project number5RM1HG009490-09
Project IDNIH-11123231 on NIH RePORTER

Who could benefit from this research

Could help

People with genetic conditions or cancers linked to specific DNA changes who can provide tissue samples or join related clinical protocols would be the most relevant candidates.

Less likely to help

Patients whose conditions are not driven by identifiable genetic changes or who require immediate standard treatments are unlikely to see direct benefit from this research in the short term.

What this research studies

This center will create advanced gene-editing and gene‑control tools (using CRISPR, engineered zinc fingers, and TALE proteins) that avoid cutting both strands of DNA to reduce damage. Teams will also develop ways to record genetic changes inside individual cells and to edit both nuclear and mitochondrial genomes. The work includes testing these tools in cells and tissues tied to human diseases, with connections to breast cancer biology such as BCAR1. Over time the technologies could support development of precise therapies, diagnostics, or clinical studies.

Why it matters

Potential benefit: If successful, these technologies could enable safer and more precise treatments or diagnostics for people with genetic disorders and certain cancers.

How similar studies have gone: Related CRISPR-based therapies and newer base/prime editors have shown early clinical promise, but many precision recording and break-free editing methods remain experimental.

Where this research is happening

  • Whitehead Institute for Biomedical Res
    Cambridge, United States
    Active

Researchers

  • Principal investigator: Weissman, Jonathan S. — Whitehead Institute for Biomedical Res
  • Study coordinator: Weissman, Jonathan S.

Related research

Other NIH-funded projects on the same topics.

  • Illuminating the dark proteome to fight cancer

    A research program aims to map and understand mysterious dark proteome proteins in cancer and to develop pre-clinical therapies that target them.

    NIH research Whitehead Institute for Biomedical Res · Cambridge, United States · NIH-11516315

  • Center for Genome Editing and Recording: Next-Generation Genome and Epigenome Tools for Disease

    A research center is developing ultra-precise genome and epigenome editing tools to better understand and treat human diseases, with a focus on how genetic variants influence disease and how to safely modify them.

    NIH research Whitehead Institute for Biomedical Res · Cambridge, United States · NIH-11412455

  • Understanding how polyamines work in human cells (a potential cancer focus)

    Researchers are studying how small molecules called polyamines help cancer-related cell growth and survival, with new ways to measure them inside living cells.

    NIH research Whitehead Institute for Biomedical Res · Cambridge, United States · NIH-11398143

  • Delivering therapeutic macromolecules with cell-derived bioparticles

    A researcher aims to safely and efficiently deliver big therapeutic molecules into human cells using cell-derived bioparticles to help treat diseases.

    NIH research Whitehead Institute for Biomedical Res · Cambridge, United States · NIH-11382122

  • Stem cell and regeneration rules in planarians

    Researchers study how planarians regulate stem cells to regrow body parts, hoping to reveal ideas that could someday help human tissue repair.

    NIH research Whitehead Institute for Biomedical Res · Cambridge, United States · NIH-11349754

  • Post-transcriptional gene regulation in human cells

    A grant studying how microRNAs control which genes are turned on or off in human cells, with a focus on cancer and development.

    NIH research Whitehead Institute for Biomedical Res · Cambridge, United States · NIH-11329480

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page.
Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.