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New Platform May Someday Treat A Variety Or Genetic Diseases.

DUARTE, Calif, July 16, 2018 -- Scientists here have discovered a gene-editing technology that could efficiently and accurately correct the genetic defects that underlie certain diseases.

The editing platform could serve as the basis for a new generation of genetic therapies used to cure inherited and acquired diseases, including sickle cell disease, hemophilia and other genetic disorders.

A City of Hope scientist's gene editing platform provides a new tool for the precise correction of genetic mutations in this rapidly growing field. It can be thought of as swapping out a mutated gene for a healthy gene to correct genetic mutations.

The genome-editing platform, tested using human blood and tissue as well as in preclinical models, is based on a family of non-disease-causing viruses known as adeno-associated viruses (AAV).

Although injecting viruses into humans may sound alarming, a large portion of the population already has been exposed to AAV with no harmful

consequences in their normal life.

The researchers isolated a subgroup of AAV known as AAVHSCs, which originate from human blood stem cells. AAVHSCs have the ability to efficiently deliver corrective DNA sequences to the nuclei of targeted cells in the body.

Through a process called homologous recombination, these corrective sequences replace disease-causing genetic mutations in the genome. Since the therapeutic correction is at the genome level, it should lead to lifelong correction.

AAVHSC-based editing vectors can efficiently edit the genome following a single administration. The researchers hope to use these properties to develop widespread and accessible genome editing used to treat genetic diseases around the world.

The editing platform works efficiently in stem cells and mature cells, including adult liver and muscle cells. Successful utilization of AAV has the potential to change the world of gene editing.

Scientists at City of Hope are attempting to build the foundation for another landmark treatment, like synthetic human insulin.

"The potential of altering the course of genetic diseases is immense," said researcher Yuman Fong. "Pairing the right AAV with blood stem cells is going to be an instrumental technique for precision medicine, the next frontier of medical treatment."

The researchers still need to characterize how this platform works and to develop it into therapeutics. They will address these questions in future studies.

Citation: Laura J. Smith et al., Stem cell-derived clade F AAVs mediate high-efficiency homologous recombination-based genome editing. Proceedings of the National Academy of Sciences, 2018; 201802343, DOI: 10.1073/pnas.1802343115


Contact: Saswati Chatterjee,

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Title Annotation:Advanced Stem Cell Technology
Publication:Stem Cell Business News
Date:Aug 6, 2018
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