Gene therapy offers fresh hope to thousands of people with blood disorders, reports a new study. The findings of the study are published in the journal Science Advances.
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.
Sickle Cell Anemia
Sickle cell anemia (SCA) is a genetic blood disorder caused by abnormal inherited hemoglobin. Sickle cell anemia is the most common form of sickle cell disease.
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‘Gene therapy can offer a potential treatment for blood disorders by engineering microparticles that deliver gene-regulating material to hematopoietic stem and progenitor cells. With further development, this technology could be useful in treatment for inherited blood disorders such as sickle cell anemia and thalassemia.’
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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 bloodstream, 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.
![Thalassemia]( "Thalassemia")
Thalassemia is an inherited blood disorder passed on through parental genes causing the body to produce abnormal 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 bloodstream: 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.
Source: Eurekalert
Thalassemia
Thalassemia is an inherited blood disorder passed on through parental genes causing the body to produce abnormal hemoglobin.
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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 bloodstream: 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.
Source: Eurekalert
In Vivo Gene Editing Cures Blood Disorders in Fetus
Monogenic hereditary disorders are largely responsible for child mortality and morbidity. However, intrauterine genetic editing could correct the genetic defect and thereby cure the disease in the fetus.
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 Self-Renewing Hematopoietic Stem Cells: Major Game-changer In Blood Disorder TherapyGeneration of self-renewing hematopoietic stem cells (HSC) is a major stride ahead in offering a potential cure for of several inherited and acquired blood diseases. | Advertisement
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