Checkpoint blockade meets gene therapy: Opportunities to improve response and reduce toxicity

Silva-Pilipich, Noelia; Covo-Vergara, Ángela; Vanrell, Lucía; Smerdou, Cristian

doi:10.1016/bs.ircmb.2023.05.006

Cited by 1 publication

(1 citation statement)

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“…58 In addition to questions regarding their toxicity, AAVs are often cleared by preexisting immunity. 59,60 Immune system avoidance will be necessary for AAVs to be effective agents for RNA therapies.…”

Section: Rna Therapeutic Deliverymentioning

confidence: 99%

RNA Therapeutics for the Cardiovascular System

Dzau,

Hodgkinson

2024

Circulation

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RNA therapeutics hold significant promise in the treatment of cardiovascular diseases. RNAs are biologically diverse and functionally specific and can be used for gain- or loss-of-function purposes. The effectiveness of mRNA-based vaccines in the recent COVID-19 pandemic has undoubtedly proven the benefits of an RNA-based approach. RNA-based therapies are becoming more common as a treatment modality for cardiovascular disease. This is most evident in hypertension where several small interfering RNA–based drugs have proven to be effective in managing high blood pressure in several clinical trials. As befits a rapidly burgeoning field, there is significant interest in other classes of RNA. Revascularization of the infarcted heart through an mRNA drug is under clinical investigation. mRNA technology may provide the platform for the expression of paracrine factors for myocardial protection and regeneration. Emergent technologies on the basis of microRNAs and gene editing are tackling complex diseases in a novel fashion. RNA-based gene editing offers hope of permanent cures for monogenic cardiovascular diseases, and long-term control of complex diseases such as essential hypertension, as well. Likewise, microRNAs are proving effective in regenerating cardiac muscle. The aim of this review is to provide an overview of the current landscape of RNA-based therapies for the treatment of cardiovascular disease. The review describes the large number of RNA molecules that exist with a discussion of the clinical development of each RNA type. In addition, the review also presents a number of avenues for future development.

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Section: Rna Therapeutic Deliverymentioning

confidence: 99%