Researchers at the Salk Institute for Biological Sciences are looking a promising approach of using stem cells to treat hemophilia.
Patients with Hemophilia B experience uncontrolled, and sometimes life threatening, bleeding due to loss or improper function of Factor IX (FIX), a protein involved in blood clotting. There is no cure for the disease and patients rely on routine infusions of FIX to prevent excessive blood loss. As you can imagine, this treatment regimen is both time consuming and expensive, while also becoming less effective over time.
Salk researchers, partially funded by CIRM, aimed to develop a more long-term solution for this devastating disease by using the body’s own cells to fix the problem.
In the study, published in the journal Cell Reports, They harvested blood cells from hemophiliacs and turned them into iPSCs (induced pluripotent stem cells), which are able to turn into any cell type. Using gene editing, they repaired the iPSCs so they could produce FIX and then turned the iPSCs into liver cells, the cell type that naturally produces FIX in healthy individuals.
One step therapy
Restoring FIX activity to this level in people with hemophilia greatly reduces bleeding rates. The treated cells also survived and produced FIX in mice for up to a year.
In a news release , first author Suvasini Ramaswamy said this approach could eliminate repeated treatments and avoid the immunosuppressive therapy required for a full liver transplant. “A cell‑based approach minimizes how often a patient needs treatment. Instead of constant injections, you can do this in one shot.”
While the results are promising, much more work is needed before this treatment can reach patients. Still, the study offers a powerful proof of principle: combining stem cell reprogramming with genetic engineering can produce transformative therapies for currently incurable genetic diseases.
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Genome Editing/Genome Engineering technique are used for insertion, deletion or modify genome of a microorganism. Techniques like TALEN, CRISPR (clustered regularly interspaced palindromic repeat) and other technique are widely used in genome editing/engineering process. CRISPR-Cas9 technique is one of the most widely preferred technologies in genome editing.
The global genome editing/genome engineering market is driven by technological advancements in the genomics, rising number of cancer patients, and increasing funding by government & private players in the genomics are major factors driving the market. However, ethical issue related to genome editing/genome engineering is likely to restrain the market to certain extent.
Read more: https://www.marketindustryreports.com/genome-editing-genome-engineering-market/5