Crispr

[Pearl]

I have seen articles about CRISPR, a gene-editing technique, and I wonder if anyone is aware of advances in the use of the technique for MDS or related diseases that would be of interest to anyone at this forum.

[Marrowforums]

The AAMDSIF Medical Advisory Board has provided an answer to your question:

CRISPR technology is a revolutionary tool that allows for efficient gene editing in a wide variety of cells. It has become invaluable in research where it can be used to make disease models in the lab. Experts have used this technique to make cancer cell lines that carry the same mutations they see in patients with MDS. They can then see how these MDS mutations change cells and how they might be targeted with specific treatments.

Examples:

Stage-specific human induced pluripotent stem cells map the progression of myeloid transformation to transplantable leukemia

A new CRISPR-engineered cancer model to test therapeutics

The clinical applications of CRISPR are appropriately less further along as the safety of using this technique in patients is being studied. That said, there are several clinical trials that use CRISPR to treat diseases, including cancer, that are already open to enrollment.

In most cases the target of CRISPR is not the tumor cells as it is very difficult to make sure that every single cancer cell gets edited. Instead, the CRISPR technique has been used to modify immune cells to make them more reactive or able to recognize specific target cells like those that form tumors.

These kinds of therapies are not yet well developed for the treatment of MDS although they are in the works. There is no doubt that CRISPR techniques will become more widespread in development and delivery of anti-cancer therapies, including those designed to treat patients with MDS.

[Pearl]

I think I see in the first 2017 article that "genetic manipulation" was shown to "return" a disease process to an earlier stage.

How long does it usually take to move these prospective findings to actual clinical practice?

[Marlene]

Looks like it's still years away but it looks like many on working on different aspect of it which is a good way to move it forward quicker.

https://www.npr.org/sections/health-...s-get-underway