Question

A researcher is studying Hutchinson Gilford Progeria Syndrome (HGPS)

He knows that this automosomal dominant disease is caused by a mutation in the LMNA gene on chromosome 1.

He also knows about crispr cas9 which can be used for gene editing

He asks you, "How can I use crispr cas9 to cure HGPS given that I would have to target every cell."

Answer his question and be sure to address how gene therapy can be used to cure a disease that affects all cell types.

Answer 1

ANS-

Hutchinson-Gilford progeria syndrome is a genetic condition characterized by the dramatic, rapid appearance of aging beginning in childhood.

Mutations that cause Hutchinson-Gilford progeria syndrome result in the production of an abnormalversion of the lamin A protein. The altered protein makes the nuclear envelope unstable and progressively damages the nucleus, making cells more likely to die prematurely.

A: CRISPR “spacer” sequences are transcribed into short RNA sequences (“CRISPR RNAs” or “crRNAs”) capable of guiding the system to matching sequences of DNA. When the target DNA is found, Cas9 – one of the enzymes produced by the CRISPR system – binds to the DNA and cuts it, shutting the targeted gene off.

When the CRISPER target for Lamin A gene , then in all cells the abnormal protein production will be shorted down.