Mice engineered with exact human DNA to model organ diseases
Genomically rewritten and tailored humanized mouse models for various organ disorders
['FUNDING_OTHER'] · NEW YORK UNIVERSITY SCHOOL OF MEDICINE · NIH-11324950
This project creates mice that carry exact human Alzheimer-related DNA so researchers can develop and test gene-based treatments for people with Alzheimer's.
Quick facts
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | NEW YORK UNIVERSITY SCHOOL OF MEDICINE (nih funded) |
| Locations | 1 site (NEW YORK, UNITED STATES) |
| Trial ID | NIH-11324950 on ClinicalTrials.gov |
What this research studies
Researchers will synthesize long stretches of human DNA from disease-linked regions and insert those human gene regions into mice to make 'GREAT-GEMMs' that more closely mimic human disease genetics. The team will use mouse embryonic stem cells to build animals carrying 50–300+ kilobase human constructs at defined genome locations. These humanized mice will be used to try therapies that need exact human sequences, such as gene therapies, CRISPR edits, and allele-specific oligonucleotides. The goal is to create a shared resource that speeds up testing of sequence-specific treatments for Alzheimer's and other organ disorders.
Who could benefit from this research
Good fit: People with Alzheimer’s disease or those known to carry specific Alzheimer-linked genetic variants are the most likely eventual beneficiaries of therapies developed using these models.
Not a fit: Patients seeking immediate symptom relief or those whose disease is unrelated to specific human genetic variants are unlikely to receive direct benefit from this laboratory-focused work.
Why it matters
Potential benefit: If successful, this could speed development and improve accuracy of gene-based treatments for Alzheimer's and related conditions.
How similar studies have performed: Humanized mouse models have informed therapy development before, but using very large synthetic human loci (50–300+ kb) to create 'GREAT-GEMMs' is a newer and less-tested approach.
Where this research is happening
NEW YORK, UNITED STATES
- NEW YORK UNIVERSITY SCHOOL OF MEDICINE — NEW YORK, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: BOEKE, JEF D — NEW YORK UNIVERSITY SCHOOL OF MEDICINE
- Study coordinator: BOEKE, JEF D
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Conditions: Alzheimer disease dementia