RESEARCH: CANCER
FOLDING PROJECT #15201 PROFILE

This project studies how PTCH1, a protein that controls cell growth, moves cholesterol around. When PTCH1 doesn't work right, it can lead to cancers like medulloblastoma and basal cell carcinoma. Understanding how PTCH1 works could help develop new cancer treatments.

This project aims to elucidate the cholesterol transport mechanism in PTCH1, a transmembrane protein responsible for inhibiting SMO in the absence of SHH in the hedgehog signaling pathway by reducing access to ciliary membrane cholesterol.

Dysregulation of this pathway (such as the inability of PTCH1 to transport cholesterol and thereby inhibit SMO) has been linked to various cancers, including medulloblastoma and basal cell carcinoma.

Thus, understanding the mechanisms behind PTCH1 cholesterol transport will allow for therapeutic treatments to target these cancers.

PTCH1

Patched 1, a protein involved in the hedgehog signaling pathway.

PTCH1 is a transmembrane protein that plays a crucial role in regulating the hedgehog signaling pathway. It acts as a receptor for Sonic Hedgehog (SHH) and inhibits SMO activity in the absence of SHH. Dysregulation of PTCH1 function has been implicated in various cancers.

SMO

Smoothened, a protein involved in the hedgehog signaling pathway.

SMO is another key player in the hedgehog signaling pathway. It acts downstream of PTCH1 and is activated by SHH binding to PTCH1. SMO activation leads to the expression of target genes that promote cell proliferation, differentiation, and survival.

SHH

Sonic Hedgehog, a signaling molecule involved in embryonic development and tissue homeostasis.

SHH is a secreted protein that acts as a ligand for the PTCH1 receptor. It plays a crucial role in regulating various cellular processes, including cell proliferation, differentiation, and migration. SHH signaling is essential for embryonic development and tissue maintenance.

Hedgehog Signaling Pathway

A complex signaling pathway involved in cell growth, differentiation, and tissue development.

The hedgehog signaling pathway is a crucial regulatory system that controls various cellular processes. It involves the interaction of proteins like PTCH1, SMO, and SHH. Disruptions in this pathway can lead to developmental abnormalities and diseases such as cancer.

Medulloblastoma

A type of malignant brain tumor that arises from the cerebellum.

Medulloblastoma is an aggressive childhood brain cancer originating in the cerebellum. This tumor can spread quickly and affect surrounding brain areas. Treatment typically involves surgery, radiation therapy, and chemotherapy.

Basal Cell Carcinoma

A common type of skin cancer that arises from basal cells.

Basal cell carcinoma is the most prevalent form of skin cancer. It usually appears as a pearly or waxy bump and often occurs on sun-exposed areas like the face. Treatment options include surgery, radiation therapy, and topical medications.