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Mesenchymal Stem cells (MSCs) derived from mouse muscle tissue and bone marrow were used in an experimental cell therapy for Duchenne Muscular Dystrophy.

Duchenne muscular dystrophy (DMD) is an incurable, progressively debilitating muscular-skeletal and cardiac disease caused by a mutation in the dystrophin gene. The encoded dystrophin protein is part of a larger protein complex involved in anchoring the muscle cytoskeleton to components of the extracellular matrix. Loss of dystrophin leads to muscle wasting, and heart disease is a major cause of death in patients with DMD. Therefore, the availability of effective treatments to address cardiac function in people with DMD is vital.

HemaCare Corporation’s latest publication in BioPharm International ^[1] sheds light on a topic that has been preoccupying some of the best minds in cell therapy research; the need to ensure a reliable supply of top-notch starting materials for up-and-coming cell and gene therapies.

Unlike traditional medical treatments, the raw materials for these therapies cannot simply be manufactured. Instead, the supply of starting materials relies on a steady influx of voluntary donors willing to take the time to undertake a fairly complex screening process and donate peripheral blood or bone marrow for altruistic reasons. Nor is a reliable donor pool the only criteria necessary to supplying materials for a successful cellular therapeutic. Starting material quality and consistency ultimately determine the reliable efficacy of the final product.

While there is no cure for acute kidney injury, a research team out of the University of Wisconsin-Madison may be shedding some new light on this condition.

Acute kidney injury (AKI) is characterized by a sudden decline in kidney function. This decline is marked by a significant rise in serum creatinine levels that may be accompanied by a reduction in urine output. Some cases of AKI require kidney transplantation or can lead to death. There are a number of causes of AKI, including injury from gadolinium-based contrast agents (nephrogenic systemic fibrosis), rhabdomyolysis, low blood volume, and vasculitis.

It’s hard to believe that 2020 is almost here! As 2019 draws to a close, it’s a good time to look back on a year abundant in innovative medical discoveries and advances. To highlight some of the most interesting developments in the cell therapy field, we are pleased to present HemaCare’s 10 most popular blogs of the year, and the improvements in healthcare that they represent. Enjoy the countdown!

CAR T cell therapy has been one of the most exciting medical advances of this decade. As a type of immunotherapy, CAR T treatment consists of collecting patient-derived immune T cells, and genetically engineering them to recognize and fight invasive cancer cells. The first of these therapies to receive U.S. Food and Drug Administration (FDA) approval, Novartis’ Kymriah^®, and Gilead’s Yescarta^® garnered international attention for their astonishing success rates in clinical trial.