Cell therapy weekly: animal study demonstrates gene therapy potential for treating rare genetic childhood disorder

Written by RegMedNet
Basic research Cell therapy Disease modeling Gene editing News News round-up

This week: researchers shed new light on the controversial existence of ‘egg stem cells’ and AMSBIO unveils a three-step kit for obtaining high yields of skeletal muscle stem cells.

The news highlights:

Gene therapy offers hope for treating rare genetic childhood disease, mouse study suggests
Do ‘egg stem cells’ exist?
Novel kit for obtaining high yields of skeletal muscle stem cells

Gene therapy offers hope for treating rare genetic childhood disease, mouse study suggests

Researchers from Imperial College London (UK) have tested a novel gene-replacement therapy in mice and human models of CDKL5 disorder — a severe neurodevelopmental condition that results from mutations in CDKL5. The disorder, which predominantly affects women, manifests in severe neurological symptoms including epilepsy and motor deficits. The results of the study, which demonstrate amelioration of disorder symptoms, suggest the therapy could offer hope for treating the rare condition.
In the study, the authors concluded: “This study provides the first evidence that gene therapy mediated by [adeno-associated virus] vectors can be used for treating CDKL5 disorder.”

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Do ‘egg stem cells’ exist?

Published in Nature Communications, investigators from the Karolinska Institutet (Stockholm, Sweden) detail their use of single-cell analytical methods to analyze over 24,000 cells harvested from ovarian cortex and medulla samples, to build a complete cell map of the human ovary. Utilizing the model, the researchers determined that the hotly debated ‘egg stem cells’, or oogonial stem cells, do not exist, which could have implications for the treatment of infertility.

Senior study author Pauliina Damdimopoulou (Department of Clinical Science, Intervention and Technology at Karolinska Institutet) commented: “The lack of knowledge about what a normal ovary looks like has held back developments [in infertility and treatment research]. This study now lays the ground on which to produce new methods that focus on the egg cells that already exist in the ovary. This could involve letting egg cells mature in test tubes or perhaps developing artificial ovaries in a lab.”

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Novel kit for obtaining high yields of skeletal muscle stem cells

AMS Biotechnology (AMSBIO; Oxford, UK) has unveiled a new kit for rapidly obtaining high yields of skeletal muscle stem cells from human pluripotent stem cells. The pluripotent stem cells transition into satellite-like or progenitor cells before ultimately fusing into myotubes, in the three-step process. The protocol could revolutionize the study of muscular diseases and therapies, which previously relied on invasive biopsy procedures.

Jason Doles, Head of the Skeletal Muscle Wasting and Progenitor Cell Biology Lab at Mayo Clinic (MN, USA), stated: “The myotube differentiation kit from AMSBIO is simple to use and it has produced consistent results every time.”

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For more weekly cell therapy news, read previous editions of the cell therapy weekly.

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