Meeting report: genetics and genome engineering

Kee, Yun; Kim, Nam‐Soo

doi:10.1007/s13258-013-0109-1

Cited by 1 publication

(2 citation statements)

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“…Transplantation of healthy NSCs to RTT patients could differentiate to generate neurons and glial cells, which could provide therapeutic benefits in RTT patients. In the in vivo study, the designed CRISPR‐Cas9 system could be texted in the zebrafish model 114–116 . Upon successful preclinical validation, therapeutic and toxicity should be evaluated in clinical trials, which, hopefully, could be translated to patients based on the consistent and positive outcomes of clinical trials.…”

Section: Disease Modelsmentioning

confidence: 99%

“…In the in vivo study, the designed CRISPR-Cas9 system could be texted in the zebrafish model. [114][115][116] Upon successful preclinical validation, therapeutic and toxicity should be evaluated in clinical trials, which, hopefully, could be translated to patients based on the consistent and positive outcomes of clinical trials.…”

Section: Gene and Stem Cell Therapymentioning

confidence: 99%

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Zebrafish in understanding molecular pathophysiology, disease modeling, and developing effective treatments for Rett syndrome

Pramanik,

Bala,

Pradhan

2024

The Journal of Gene Medicine

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Rett syndrome (RTT) is a rare but dreadful X‐linked genetic disease that mainly affects young girls. It is a neurological disease that affects nerve cell development and function, resulting in severe motor and intellectual disabilities. To date, no cure is available for treating this disease. In 90% of the cases, RTT is caused by a mutation in methyl‐CpG‐binding protein 2 (MECP2), a transcription factor involved in the repression and activation of transcription. MECP2 is known to regulate several target genes and is involved in different physiological functions. Mouse models exhibit a broad range of phenotypes in recapitulating human RTT symptoms; however, understanding the disease mechanisms remains incomplete, and many potential RTT treatments developed in mouse models have not shown translational effectiveness in human trials. Recent data hint that the zebrafish model emulates similar disrupted neurological functions following mutation of the mecp2 gene. This suggests that zebrafish can be used to understand the onset and progression of RTT pathophysiology and develop a possible cure. In this review, we elaborate on the molecular basis of RTT pathophysiology in humans and model organisms, including rodents and zebrafish, focusing on the zebrafish model to understand the molecular pathophysiology and the development of therapeutic strategies for RTT. Finally, we propose a rational treatment strategy, including antisense oligonucleotides, small interfering RNA technology and induced pluripotent stem cell‐derived cell therapy.

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Section: Disease Modelsmentioning

confidence: 99%

Section: Gene and Stem Cell Therapymentioning

confidence: 99%