Gene Therapy for SADS: Is this the New Holy Grail? (Joint Session)-Gene Therapy for SADS: Is this the New Holy Grail? (Joint Session)

Video Transcription

Video Summary

The session centered on the rapid advancements and applications of gene therapy and its potential role in treating cardiovascular diseases, specifically in SADS conditions. Speakers acknowledged the unprecedented growth in gene therapy options, noting there were 33 approved gene therapies, along with thousands in pre-FDA registration stages, mostly targeting non-cardiac diseases. The landscape for gene therapy and cell therapy was outlined, emphasizing the challenge of applying these advancements to cardiovascular diseases.<br /><br />Gene therapy's potential was highlighted, demonstrating developments in areas like RNA therapies and genome editing using technologies such as CRISPR-Cas9. Discussions explored how different gene mutations require unique therapeutic approaches—for example, gene replacement for haploinsufficient conditions or RNA therapies for altering the effects of mutations.<br /><br />The session also addressed the challenges and side effects observed in gene therapy applications, including immune reactions and multi-organ complications, as illustrated through patient cases. This prompted discourse on the need for tailored immunosuppressant strategies to manage these side effects effectively.<br /><br />Further, the session explored non-gene therapy approaches to treating long QT syndrome, emphasizing that intentional non-treatment, monotherapy through cardiac sympathetic denervation, and existing drugs like myxilatine remain crucial until gene therapies become mainstream for this condition.<br /><br />Speakers concluded that while gene therapy holds significant promise, challenges such as ensuring targeted delivery and managing immune responses remain. They predicted that by 2035, advancements including safer delivery vectors and therapies targeting specific pathways would enhance treatment options for genetic cardiovascular conditions, though the field still requires greater educational pathways and clinical integration.

Keywords

gene therapy

cardiovascular diseases

SADS conditions

CRISPR-Cas9

RNA therapies

genome editing

immune reactions

long QT syndrome

therapeutic approaches

clinical integration