Spinal Muscular Atrophy Stem Cell Therapy Development

Stem cell therapy is considered an alternative approach to treat spinal muscular atrophy (SMA). Stem cell transplantation is a potential therapeutic strategy to alter the extracellular motor neuron environment not only through cell replacement but also through trophic and neuroprotective effects. CD BioSciences offers stem cell therapy development service for SMA, dedicated to advancing the treatment of this disease.

Overview of Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a devastating neurodegenerative disease characterized by degeneration of low-level motor neurons in the spinal cord, leading to progressive paralysis and early death in severe cases. SMA is primarily caused by mutations in the genes of the survival motor neurons (SMN). SMA is characterized by muscle weakness and atrophy due to progressive degeneration and irreversible loss of the anterior horn cells of the spinal cord (i.e., lower motor neurons) and brainstem nuclei. The onset of weakness ranges from prenatal to adult. The weakness is symmetrical, greater proximally than distally, and is progressive.

SMA is classified by different clinical severities into 4 types:

• Type I SMA is the most severe type, also known as Werdnig-Hoffman disease.
• Type II SMA is not as severe as type I. Patients usually do not develop symptoms of SMA in early infancy, but they become increasingly frail over time.
• Type III SMA is a less severe form that usually begins in early adulthood and gets progressively worse as the patient ages.
• Type IV SMA is the least severe type of the disease and usually begins to show weakness and muscle atrophy in adulthood.

Stem Cell Therapy for Spinal Muscular Atrophy

Stem cell therapy is a promising approach for treating SMA, as it offers the potential to replace the missing or defective motor neurons that are responsible for the disease. There are several types of stem cells that can be used for SMA therapy, including embryonic stem cells, induced pluripotent stem cells, and mesenchymal stem cells. These stem cells can be differentiated into motor neurons and transplanted into the spinal cord to replace the missing or defective cells.

Therapeutic properties of mesenchymal stromal cells (MSCs) in muscle injury (Sandonà, Martina et al., 2021)

In addition to cell replacement, stem cells can also be engineered to produce SMN protein or other therapeutic agents that can improve motor neuron survival and function. For example, stem cells can be modified to express genes that increase SMN production or to produce neurotrophic factors that promote motor neuron growth and survival.

Our Services

Stem cell therapy is a promising treatment for SMA that aims to replace the lost motor neurons and improve muscle function. CD BioSciences offers stem cell therapy development services for SMA. Based on our advanced technologies and years of experiences, we are committed to advance the development of stem cell therapy for SMA. Our services are included but not limited as follows:

As a pioneer in biotechnology, CD BioSciences has grown into one of the largest independent biotechnology companies in the world. CD BioSciences is committed to providing professional and efficient service to our customers around the world. If you are interested in our service, please contact us.

Reference

1. Sandonà, Martina et al. "Mesenchymal Stromal Cells and Their Secretome: New Therapeutic Perspectives for Skeletal Muscle Regeneration." Frontiers in bioengineering and biotechnology vol. 9 652970. 13 May. 2021.