Article

Biotechnological Challenges in STXBP1 Syndrome Research: Gene and Cell Therapy, Experimental Models and Future Clinical Perspectives

2024-12-20

Abstract excerpt

STXBP1 syndrome is a severe early-onset epileptic encephalopathy characterized by developmental delay and intellectual disability. This review addresses key challenges in STXBP1 syndrome research, focusing on advanced therapeutic approaches and experimental models. We explore gene therapy strategies, including CRISPR-Cas9, adeno-associated viral (AAV) vectors, and RNA therapies such as antisense oligonucleotides (...

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Literature Corpus work: 010a293f-01c3-550a-a351-f362c0a994e6
DOI: 10.20944/preprints202412.1685.v1

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