Developing Protocols for Modelling of Genetic Diseases Using Induced Pluripotent Stem Cell
Recent advances have shown that cells from human blood, skin and urine samples can be reprogrammed to become stem cells. These are called induced Pluripotent Stem Cells (iPSCs) and share many characteristics with embryonic stem cells, including an unlimited capacity for proliferation and the potential to become any cel
| Condition(s) | Genetic Disease |
|---|---|
| Status | Recruiting |
| Study type | Observational |
| Summary | Recent advances have shown that cells from human blood, skin and urine samples can be reprogrammed to become stem cells. These are called induced Pluripotent Stem Cells (iPSCs) and share many characteristics with embryonic stem cells, including an unlimited capacity for proliferation and the potential to become any cell in the body. Beneficially, the use of iPSCs avoids the ethical difficulties which surround embryonic stem cells and allows generation of iPSC lines which are disease representative. For example, we could take skin samples from an individual diagnosed with Huntington's disease and their unaffected sibling and using this technology, generate iPSC lines from both individuals. Using these iPSCs, we could produce disease affected cell populations from the affected and unaffected |
| Who can participate | Inclusion Criteria: * Male or female * Individuals diagnosed with a genetic disease - any age between 1-120 years. * Individuals diagnosed with a genetic disease - must be able to communicate well with the investigator and to comply with the requirements of the entire study OR be under the care of an appropriate guardian, if incapacitated or under the age of 16. * Individuals diagnosed with a genetic disease - require provision of written informed consent either by participant or guardian, to participate as shown by a witnessed signature on the Subject Consent Form * Individuals participating as controls - aged between 16-120 years. * Individuals participating as controls -must be able to consent for themselves Exclusion Criteria: * Individuals less than 1 year old. * Infection with any bl |
| Ages | 1 Year to 120 Years |
| Sex | All |
| Lead sponsor | Sapna Vyas |
| Locations | Edinburgh, Midlothian, United Kingdom |
| Start date | 2018-11-01 |
| NCT ID | NCT03612310 |
| Official listing | https://clinicaltrials.gov/study/NCT03612310 |