Research

Life Sciences & Biotechnology

Title :

Synergistic Approaches for Spinal Cord Injury Repair: Combined Gene Therapy and Stem Cell Transplantation

Area of research :

Life Sciences & Biotechnology

Focus area :

Gene Therapy, Stem Cell Therapy

Principal Investigator :

Dr. Shringika Soni, CSIR- Centre For Cellular And Molecular Biology (CSIR–CCMB), Hyderabad, Telangana

Timeline Start Year :

2024

Timeline End Year :

2026

Contact info :

Details

Executive Summary :

Spinal nerves are crucial for transmitting signals between the brain and the body, enabling body movements and balance. However, in cases of Spinal Cord Injury (SCI), the regenerative capacity of spinal nerves becomes limited, leading to loss of functioning. This is due to diminished intrinsic regenerative ability within neurons and extrinsic inhibitors that impede axon regeneration. To address these limitations, a promising approach is proposed, which involves a simultaneous intervention targeting both intrinsic and extrinsic barriers. The study aims to evaluate the effects of gene therapy involving anterograde and retrograde delivery of pro-growth factors, namely Nr5a2 and Rarb transcription factors, along with stem cells to promote robust axon growth in injured mice. Thoracic crush injury (TCI) will be performed to create SCI injury in animals, and the effect of Nr5a2/Rarb-AAV-based gene and neural stem cell therapy will be evaluated through molecular and behavioral assays. The grafting stage of stem cells for significantly effective neural regeneration will be validated. The study will also conduct motor function tests to assess functional recovery in the injured animals and correlate it with specific neural markers at the molecular level. Genomics techniques will investigate gene expression profiles and molecular changes associated with the delivery of Nr5a2/Rarb and stem cell implantation, allowing for the elucidation of specific pathways and molecular interactions involved in promoting axon growth and regenerative processes. Advanced imaging techniques and histological analyses will also be used to assess axon morphology, connectivity, and regenerative capacity in the treated animals.

Organizations involved