The promise of episomal gene therapy

KOSTOHRYZ, Viktor; Jiří KOHOUTEK a Ondřej SLABÝ. The promise of episomal gene therapy. In 6th CCP Phenogenomics Conference 2024. 2024. ISBN 978-80-907896-5-4.

Základní údaje

Originální název

The promise of episomal gene therapy

Autoři

KOSTOHRYZ, Viktor ORCID; Jiří KOHOUTEK a Ondřej SLABÝ

Vydání

6th CCP Phenogenomics Conference 2024, 2024

---

Další údaje

Typ výsledku

Konferenční abstrakt

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Označené pro přenos do RIV

Ne

ISBN

978-80-907896-5-4

---

Anotace

V originále

Modern day gene therapies are limited by a number of shortcomings that limit their practical use in the treatment of many key genetic diseases. The most significant limitations include 1) high immunogenicity of the delivery vector, 2) low stability in target cells and 3) mutagenicity of the construct. The answer to the aforementioned shortcomings is episomal gene therapy. Episomes are synthetic plasmid constructs containing regulatory sequences from mammalian chromatin. These sequences, the so-called "S/MARs", allow plasmids to stably segregate into daughter cells throughout many cell divisions, and help them prevent integration into the genome by a currently unknown mechanism (1). However, the stability of episomes in mammalian cell lines without selection is very low and currently requires significant improvement for further practical use. This problem goes hand in hand with the fact that the mechanisms of episomal retention and resistance to integration still remain poorly understood.