Nav: Home

Gene therapy for blood disorders

December 04, 2018

Gene therapy holds a lot of promise in medicine. If we could safely alter our own DNA, we might eliminate diseases our ancestors passed down to us.

Now, a team of University of Delaware researchers has demonstrated a major step forward in gene therapy by engineering microparticles that deliver gene-regulating material to hematopoietic stem and progenitor cells, which live deep in our bone marrow and direct the formation of blood cells. In a paper published in the journal Science Advances, Chen-Yuan Kao, a doctoral student in chemical engineering, and Eleftherios T. (Terry) Papoutsakis, Unidel Eugene du Pont Chair of Chemical and Biomolecular Engineering, describe how they used megakaryocytic microparticles, which circulate naturally in the blood stream, to deliver plasmid DNAs and small RNAs to hematopoietic stem cells.

With more development, this technology could be useful in treatment for inherited blood disorders that affect thousands of Americans. These include, for example, sickle cell anemia, a disease that causes abnormally shaped red blood cells, and thalassemia, which disrupts the production of the blood protein hemoglobin.

The methods developed by Kao and Papoutsakis could also be used to deliver personalized medicine, because these microparticles can be individually generated and stored frozen for each patient, said Papoutsakis.

This novel approach has advantages over other approaches under investigation.

"A lot of researchers are trying to deliver DNA, nucleic acids, or drugs to target hematopoietic stem cells," said Papoutsakis. "This is the right cell to target because it gives rise to all blood cells."

Alter those cells, and you could, in theory, ward off the genetic defect for most or all of the patient's life.

However, some previously developed methods to target these stem cells deliver genetic material with help from a virus, risking side effects to the patient, said Papoutsakis. Instead, the University of Delaware team developed a method that takes advantage of tiny particles that already float in the blood stream: megakaryocytic microparticles. Kao and Papoutsakis found that they could load these microparticles with gene-regulating material and that they would infiltrate only the desired stem cells, thanks to special properties on the surface of the microparticles.
-end-
Next up: Papoutsakis is collaborating with Emily Day, assistant professor of biomedical engineering at UD, to explore another way to deliver this material. Day, an expert in nanomedicine, won the NSF CAREER award earlier this year to study membrane-wrapped nanoparticles for gene therapy.

University of Delaware

Related Gene Therapy Articles:

Restoring vision by gene therapy
Latest scientific findings give hope for people with incurable retinal degeneration.
Gene therapy/gene editing combo could offer hope for some genetic disorders
A hybrid approach that combines elements of gene therapy with gene editing converted an experimental model of a rare genetic disease into a milder form, significantly enhancing survival, shows a multi-institutional study led by the University of Pennsylvania and Children's National Hospital in Washington, D.C.
Using gene therapy to treat chronic traumatic encephalopathy
A new study shows the feasibility of using gene therapy to treat the progressive neurodegenerative disorder chronic traumatic encephalopathy (CTE).
New technology allows control of gene therapy doses
Scientists at Scripps Research in Jupiter have developed a special molecular switch that could be embedded into gene therapies to allow doctors to control dosing.
Gene therapy: Development of new DNA transporters
Scientists at the Institute of Pharmacy at Martin Luther University Halle-Wittenberg (MLU) have developed new delivery vehicles for future gene therapies.
Non-viral gene therapy to speed up cancer research
A new treatment method promises to speed up gene therapy research and could bring new, patient friendly cancer treatments to market faster.
Gene therapy promotes nerve regeneration
Researchers from the Netherlands Institute for Neuroscience and the Leiden University Medical Center have shown that treatment using gene therapy leads to a faster recovery after nerve damage.
Gene therapy for blood disorders
Delivering gene-regulating material to cells that live deep in our bone marrow and direct the formation of blood cells.
Realizing the potential of gene therapy for neurological disorders
Promising findings from preclinical animal studies show the potential of gene therapy for treating incurable neurological disorders.
Gene therapy vectors carrying the telomerase gene do not increase the risk of cancer
Researchers from the Spanish National Cancer Research Centre (CNIO) have shown in a new study that the gene therapy with telomerase that they have developed, and which has proven to be effective in mice against diseases caused by excessive telomere shortening and ageing, does not cause cancer or increase the risk of developing it, even in a cancer-prone setting.
More Gene Therapy News and Gene Therapy Current Events

Trending Science News

Current Coronavirus (COVID-19) News

Top Science Podcasts

We have hand picked the top science podcasts of 2020.
Now Playing: TED Radio Hour

Our Relationship With Water
We need water to live. But with rising seas and so many lacking clean water – water is in crisis and so are we. This hour, TED speakers explore ideas around restoring our relationship with water. Guests on the show include legal scholar Kelsey Leonard, artist LaToya Ruby Frazier, and community organizer Colette Pichon Battle.
Now Playing: Science for the People

#568 Poker Face Psychology
Anyone who's seen pop culture depictions of poker might think statistics and math is the only way to get ahead. But no, there's psychology too. Author Maria Konnikova took her Ph.D. in psychology to the poker table, and turned out to be good. So good, she went pro in poker, and learned all about her own biases on the way. We're talking about her new book "The Biggest Bluff: How I Learned to Pay Attention, Master Myself, and Win".
Now Playing: Radiolab

Uncounted
First things first: our very own Latif Nasser has an exciting new show on Netflix. He talks to Jad about the hidden forces of the world that connect us all. Then, with an eye on the upcoming election, we take a look back: at two pieces from More Perfect Season 3 about Constitutional amendments that determine who gets to vote. Former Radiolab producer Julia Longoria takes us to Washington, D.C. The capital is at the heart of our democracy, but it's not a state, and it wasn't until the 23rd Amendment that its people got the right to vote for president. But that still left DC without full representation in Congress; D.C. sends a "non-voting delegate" to the House. Julia profiles that delegate, Congresswoman Eleanor Holmes Norton, and her unique approach to fighting for power in a virtually powerless role. Second, Radiolab producer Sarah Qari looks at a current fight to lower the US voting age to 16 that harkens back to the fight for the 26th Amendment in the 1960s. Eighteen-year-olds at the time argued that if they were old enough to be drafted to fight in the War, they were old enough to have a voice in our democracy. But what about today, when even younger Americans are finding themselves at the center of national political debates? Does it mean we should lower the voting age even further? This episode was reported and produced by Julia Longoria and Sarah Qari. Check out Latif Nasser's new Netflix show Connected here. Support Radiolab today at Radiolab.org/donate.