TLI Investigator Dr. Denise Al Alam Receives $1.5 Million Grant from CIRM to Explore Genetic Defects of Lung Disease in Down Syndrome
This study is poised to generate crucial new Down Syndrome cell lines to study genetic defects specific to Down Syndrome.
Tuesday, April 2, 2024
Investigator(s)
The Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center (TLI) has announced that The California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated to regenerative medicine, has awarded $1.5 million to TLI Investigator Denise Al Alam, PhD, to support research that aims to understand lung disease in individuals with Trisomy 21 (also known as Down Syndrome). Although Trisomy 21 impacts multiple organ systems, respiratory complications are a significant cause of death in children and adults with this genetic condition.
With the highest occurrence of Down Syndrome births in California within the Latinx community, this research bears not just scientific significance but also a profoundly human one. Dr. Al Alam’s team is at the forefront of utilizing ethically sourced pluripotent stem cells (iPSCs) from a tapestry of ethnic backgrounds, pioneering a path to understand and combat the alveolar defects in Trisomy 21.
“This research is not just about understanding a condition. It’s about changing lives,” explains Dr. Al Alam. “Our work strives to be the beacon of hope for those grappling with the complexities of Trisomy 21. By transcending the limitations of existing models and forging new ones, we are on the cusp of uncovering genetic insights that could lead to transformative therapies. The future we envision is one where every breath for individuals with Trisomy 21 isn’t just possible, but effortlessly joyful.” This award is a collaborative effort with Lundquist investigator Dr. Soula Danopoulos, Dr. Cristina Maria Alvira from Stanford University, and Dr. Jessie Huang from Keck School of Medicine of USC.
This pioneering study heralds a new dawn for therapeutic innovation, offering a glimmer of hope for nearly 667 Down Syndrome newborns in California each year. The project's success could result in a shift in treatment paradigms and significantly improve the quality of life for children and adults living with Down Syndrome globally.
“This investment in Dr. Al Alam’s research represents our commitment to groundbreaking work that can forge new paths for early intervention and vastly improve living conditions for the Down Syndrome community,” said Dr. Rosa Canet-Avilés, Vice President of Scientific Programs at CIRM.
The Lundquist Institute is not just conducting research; it’s sculpting a future where medical advancements translate into tangible differences in health outcomes for one of the most vulnerable populations among us.