Rida Shaikh
Project: Making a safer, easier-to-give treatment for spinal muscular atrophy using a chain of varying amino acids bound to DNA-like molecules that reach the whole body
“In research, even unexpected findings can provide valuable clues that guide the next discovery. I have learned that asking thoughtful questions, embracing uncertainty and being open to learning from every result are what transform experiments into meaningful scientific progress. Curiosity doesn't just help us understand the world, it drives discoveries that can change it.”
Summary: Spinal muscular atrophy (SMA) is a severe inherited disease that affects infants and young children, causing progressive muscle weakness and difficulty breathing. It occurs when the body lacks a protein called survival motor neuron (SMN), which is critical for healthy nerve and muscle function.
Several treatments for SMA have been approved, including gene therapy and antisense oligonucleotides (ASOs) — small DNA-like molecules that help cells produce SMN protein. While these therapies have greatly improved survival and outcomes, important limitations remain, including incomplete response in some patients, uncertainty about long-term durability and potential side effects. The current approved ASO treatment requires repeated injections into the spinal fluid, which can be invasive and burdensome for children and families. In addition, because SMA affects multiple organs, there remains a need for treatments that can efficiently restore SMN in both the nervous system and peripheral tissues.
My research addresses this problem using DG9, a chain of amino acids called a peptide that aids delivery. After a simple injection under the skin, DG9 helps ASOs enter cells more effectively and reach tissues throughout the body. Previous work has shown that DG9-delivered ASOs improve survival and muscle function in severe SMA animal models. This project aims to identify the most effective and safest version of DG9. I will test eight DG9 variants to determine how different sizes and charges affect ASO delivery, SMN restoration, tissue distribution and therapeutic benefit in SMA animal models.
This work could support the development of safer, less invasive, whole-body treatments for children with SMA, and may inform delivery strategies for other genetic diseases.
Rida Shaikh was supervised by Dr. Toshi Yokota in the Department of Medical Genetics in the Faculty of Medicine & Dentistry. Her WCHRI summer studentship was funded by the Stollery Children’s Hospital Foundation. She is studying neuroscience at the University of Alberta.
Read more about WCHRI’s training awards for students at all levels and generous grants for researchers. See the rest of the 2026 summer studentship recipients here.
