When will stem cells save more lives?
Melissa Little and her colleagues worked for six years to bring the world’s largest stem cell meeting to Melbourne this week.
What did she learn? What are the next big steps should we should be watching for in curing diseases and saving lives with stem cells?
Melissa can also talk about her own research at the Murdoch Children’s Research Institute. She’s made mini-kidneys that are a step towards stopping a silent killer, chronic kidney disease.
The International Society for Stem Cell Research 2018 Annual Meeting closes today. 2,500+ stem cell scientists from 53 countries heard from 150+ speakers.
Treating haemophilia and eye disease with gene therapy
Katherine High (USA) will report today on an FDA approved gene therapy for a form of blindness, and on a clinical trial in people with haemophilia.
She’s the head of R and D for Spark Therapeutics, a spin-out from the Children’s Hospital of Philadelphia.
Last year, the US FDA announced the approval of the first directly administered gene therapy targeting a rare inherited form of blindness caused by mutations in a specific gene. The therapy uses a modified virus to deliver a normal copy of a defective gene directly into retinal cells in the eye. The corrected code makes the eyes cells function normally, producing the proteins they need to restore vision.
In May, Spark Therapeutics also announced progress in clinical trials of gene therapies for bleeding disorders haemophilia B and A.
Making heart muscle cells
Martin Laflamme (Canada) can make large quantities of heart muscles and regenerate muscle tissue (in animals). Now he’s working to refine the technique. The aim is to make mature, functional heart cells from stem cells and successfully transplant them into patients to repair tissue damage caused by heart attacks.
Michael is a physician-scientist at the Toronto General Hospital Research Institute.
How do melanoma and leukemia start?
Dr Leonard Zon (USA) will reveal the first steps of cancer. He has previously showed that melanoma starts with a reactivation of the stem cell program that is used to make melanocytes in embryos. Now he has turned his attention to studying the beginning of leukemia.
As people age, they accumulate mutations in specific genes in their blood. Those genes cause the stem cells to expand and this predisposes the cell to acquire mutations and over time become cancerous.
Using the zebrafish, Dr Zon’s group has modelled these early events, and can see them happening by labelling each stem cell in a different colour and watching for a colour to become dominant in the blood. That represents the early stage of leukemia and he can study it. The effects of the stem cells are often due to their environment, called the niche. Dr Zon has found a code for the blood vessels in the niche that help the stem cell migrate and divide.
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