‘Genetic doppelgangers:’ Emory research provides insight into two neurological puzzles

An international team led by Emory scientists has gained insight into the pathological mechanisms behind two devastating neurodegenerative diseases. The scientists compared the most common inherited form of amyotrophic lateral sclerosis and frontotemporal dementia (ALS/FTD) with a rarer disease called spinocerebellar ataxia type 36 (SCA 36). Both of the diseases are caused by abnormally expanded and strikingly similar DNA repeats. However, ALS progresses quickly, typically killing patients within a year or two, while the disease Read more

Emory launches study on COVID-19 immune responses

Emory University researchers are taking part in a multi-site study across the United States to track the immune responses of people hospitalized with COVID-19 that will help inform how the disease progresses and potentially identify new ways to treat it.  The study is funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health. The study – called Immunophenotyping Assessment in a COVID-19 Cohort (IMPACC) – launched Friday. Read more

Marcus Lab researchers make key cancer discovery

A new discovery by Emory researchers in certain lung cancer patients could help improve patient outcomes before the cancer metastasizes. The researchers in the renowned Marcus Laboratory identified that highly invasive leader cells have a specific cluster of mutations that are also found in non-small cell lung cancer patients. Leader cells play a dominant role in tumor progression, and the researchers discovered that patients with the mutations experienced poorer survival rates. The findings mark the first Read more

islet transplants

Challenges in islet transplantation

Two recent research papers from the Emory Transplant Center describe research on pancreatic islet transplantation, an experimental procedure that could help people with type I diabetes live without daily insulin injections.

Islet transplantation may offer people with type I diabetes the ability to produce their own insulin again

As with other types of transplantation, the challenge with islet transplantation is to avoid rejection of the donated organ and to balance that goal against side effects from the drugs needed to control the immune system. These papers illustrate how that balancing act is especially complex.

In the last decade, transplant specialists developed a method for islet transplantation named the “Edmonton protocol” after pioneers at the University of Alberta. While the emergence of this method was a major step forward, there are limitations:

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Pig stem cells: hope for Type 1 diabetes treatment

University of Georgia researchers recently reported on their work to create pigs with induced pluripotent stem cells. This type of cell, first developed about five years ago, has the ability to turn into any other kind of cell in the body.

An Emory transplant team, working with the UGA group, hopes to use this technology to develop pig islet cells as an alternative to human islets to treat patients with Type 1 diabetes. Type 1 diabetes usually occurs early in life and affects more than one million Americans who are unable to manufacture their own insulin because their pancreatic islets do not function.

Emory islet transplant team

The Emory Transplant Center has conducted clinical trials since 2003 transplanting human pancreatic islet cells into patients with Type I diabetes. Some of these patients have been able to give up insulin injections, either temporarily or permanently. Other sources of islets are needed for transplant though because of the large number of potential patients and because each transplant typically requires islets from several pancreases.

To create pigs using pluripotent stem cells, the UGA team injected new genes into pig bone marrow cells to reprogram the cells into functioning like embryonic stem cells. The resulting pluripotent cells were inserted into blastocysts (developing embryos), and the embryos were implanted into surrogate mothers. The resulting pigs had cells from the stem cell lines as well as the embryo donor in multiple tissue types.

The pluripotent stem cell process could allow researchers to make genetic changes to dampen or potentially eliminate the rejection of the pig islets by the human immune system.

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