Patient Specific Induced Pluripotency Stem Cells (PSiPS)

Royan Institute

Status: Completed

Conditions: Eye Disorders; Hepatic Disorders

Treatments: Procedure: Biopsy

Study type: Observational

Funder types: Other

Identifiers

NCT00953693

Royan-iPS-001

Details and patient eligibility

About

Induced pluripotent stem cells (iPSCs) are adult cells that have been genetically reprogrammed to an embryonic stem cell-like state by being forced to express genes and factors important for maintaining the defining properties of embryonic stem cells. The reprogramming of adult cells into embryonic stem (ES) cells enables the generation of patient-specific stem cells and thus has enormous potential for the treatment and analysis of degenerative diseases. In this project the investigators are going to induce pluripotent stem cells from cell cultures from skin biopsies of patients. The iPS cells will be developed for modeling diseases and drug discovery as well as basic research.

Full description

iPS cells are typically derived by transfection of certain stem cell-associated genes into non-pluripotent cells, such as adult fibroblasts. Transfection is typically achieved through viral vectors, such as retroviruses. Transfected genes include the master transcriptional regulators Oct-3/4 (Pouf51) and Sox2, although to enhance the efficiency of induction some other genes e.g. Klf4, c-Myc. In this study after separation the fibroblasts from patient's skin biopsy, the cells are transfected with 4 Yamanaka factors (human Oct4, Sox2, Klf4, c-Myc) after 3-4 weeks, transfected cells are isolated through morphological selection.

Enrollment: 15 estimated patients

Sex: All

Ages: 12 to 80 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

• Donors suffering from different (specified) metabolic disorders
• Donors suffering from different (specified) eye disorders which resulted to blindness
• Special blood group: O Bombay

Exclusion criteria

• None