Advancing Gene Therapies and Novel Treatment Strategies for Infiltrative Cardiomyopathies: A Comprehensive Review of Targeted Interventions - PubMed
5 hours ago
- #Cardiomyopathy
- #Gene Therapy
- #CRISPR
- Recent progress in understanding molecular mechanisms of infiltrative Cardiomyopathies (CMPs) has opened new avenues for targeted therapies.
- Conditions like cardiac amyloidosis, sarcoidosis, Danon disease, Fabry disease, Mucopolysaccharidoses (MPS), and cardiac oxalosis impair cardiac function through complex mechanisms.
- In cardiac amyloidosis, misfolded proteins form fibrillary amyloids, disrupting myocardial structure and causing inflammation, oxidative stress, and apoptosis.
- New treatments such as Antisense Oligonucleotides (ASOs), siRNA, and monoclonal antibodies show promise in managing amyloid deposition.
- CRISPR-Cas9 gene editing offers potential for lasting therapeutic benefits by correcting pathogenic mutations.
- Enzyme Replacement Therapies (ERT) and chaperone molecules improve outcomes in Fabry disease, though challenges persist in advanced stages.
- Immunomodulatory strategies and antibody-based therapies targeting protein aggregates show efficacy in preclinical and early-phase trials.
- Challenges include drug delivery efficiency, off-target effects, and inconsistent clinical responses among patient groups.
- Future research should focus on improving therapy specificity and safety to enhance patient outcomes and quality of life.