Carlos Moraes, PhD, on Understanding Mitochondrial Mutations for Neurodegenerative Diseases
The Lichtenstein professor of neurology at University of Miami Miller School of Medicine discussed research his lab is pursuing and its applications.
“Mitochondrial DNA mutations have also been associated with neurodegenerative diseases and even normal aging... When you’re older and maybe have some predisposition, and some mutated mitochondria DNA, there are several hypotheses that this contributes to neurodegenerative disease. So, mutations of mitochondria can also apply to those disorders. Parkinson disease is one example... this research has a broader appeal for therapy.”
Carlos Moraes, PhD, Lichtenstein professor of neurology, University of Miami Miller School of Medicine, and his lab are focused on mitochondrial diseases and understanding mitochondrial mutations in the mtDNA. This research could have implications for a wide variety of diseases, including neurodegenerative diseases such as Parkinson disease.
Moraes chaired a session at the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-6, in Orlando, Florida, entitled Dysfunction in Cellular Organelles and Trafficking in Neuromuscular Disorders. The session was one of a number focused on disease biology and mechanisms.
CGTLive® spoke with Moraes to learn more about mitochondrial DNA (mtDNA) and mutations. He talked about researching the potential of gene editing in mtDNA and how his lab is working with Precision Biosciences to use their gene editing platform. He discussed some diseases that this work may benefit and talked about how gene therapy is growing in the field in general. He identified delivery of adeno-associated virus vectors as one aspect that needs to be improved, especially delivery to the central nervous system. He also shared his excitement for topics that were discussed at the MDA meeting, including in vitro models of muscle organoids that may help his research.
Click here to view more coverage of the 2024 MDA Conference.
REFERENCE
Moraes C. Dysfunction in Cellular Organelles and Trafficking in Neuromuscular Disorders. Session. Presented at: 2024 MDA Clinical and Scientific Conference; March 3-6; Orlando, FL.
Considerations for Administering Hemoglobinopathy Gene Therapies From a Pharmacist’s Perspective
April 20th 2024Alexis Kuhn, PharmD, BCOP, a pediatric oncology pharmacist at Mayo Clinic, discussed the incorporation of the recently FDA-approved gene therapies for SCD and TDT into the work of pharmacists.
