The prospect of treating genetic diseases with corrected stem cells grown from patients’ own bodies has moved closer, after the results of a remarkable experiment.
Scientists have successfully reprogrammed skin tissue from people with a rare form of anaemia to create powerful stem cells, while at the same time rectifying the genetic defect that causes the condition.
The corrected stem cells could be grown into blood precursor cells for therapy. As these would carry a patient’s own DNA, except for the mutation responsible for the illness, they could be transplanted without risk of rejection by the body’s immune system.
Though the research
British regulators have given Moorfields approval to begin trials using retinal cells derived from human embryonic stem cells (hESCs).
Twelve patients with Stargardt’s disease will have the cells injected into the eye. You can read more about the trial here.
Although there is great excitement about the trial, Julia knows that the initial phase will simply check safety (…)
“It would be marvellous if I could get some of my sight-loss reversed”, said Julia. “Even if it simply halts the deterioration, that would be great. And the real benefit would be for children. It could mean they don’t need to lose any
Using two distinct methods, Whitehead Institute researchers have successfully and consistently manipulated targeted genes in both human embryonic stem (ES) cells and induced pluripotent stem (iPS) cells (adult cells that have been reprogrammed to an embryonic stem cell-like state).
In one case, scientists employed proteins known as zinc finger nucleases (ZFNs) to change a single base pair in the genome, allowing them either to insert or remove mutations known to cause early-onset Parkinson’s disease (PD). The second method relies on proteins called transcription activator like effector nucleases (TALENs) capable of altering specific genes with similar efficiency and precision as ZFNs.
Researchers at Johns Hopkins have found a better way to create induced pluripotent stem (iPS) cells—adult cells reprogrammed with the properties of embryonic stem cells—from a small blood sample. This new method, described last week in Cell Research, avoids creating DNA changes that could lead to tumor formation.
“These iPS cells are much safer than ones made with previous technologies because they don’t involve integrating foreign viruses that can potentially lead to uncontrolled, cancerous cell growth,” says Linzhao Cheng, Ph.D., an associate professor of medicine in the Division of Hematology and a
Stem-cell science is a fast-moving field. Just three years since a Japanese researcher first reprogrammed ordinary skin cells into stem cells without the use of embryos, scientists at a Massachusetts biotech company have repeated the feat, only this time with a new method that creates the first stem cells safe enough for human use. The achievement brings the potentially lifesaving technology one step closer to real treatments for disease.
Dr. Robert Lanza, chief scientific officer at Advanced Cell Technology (ACT), reported today in the journal Cell that his team has created stem cells using human skin cells and four proteins.