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Tinjauan Literatur Sistematis; Pemanfaatan Teknologi CRISPR-CAS9 Untuk Pengobatan Penyakit Huntington’s
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A Systematic Literature Review: Utilization of CRISPR-CAS9 Technology for Treating Huntington's Disease
Huntington's Disease (HD) is a neurodegenerative disorder caused by a CAG triple expansion (>36) in the first exon of the HTT gene encoding the huntingtin protein. Advances in gene editing technologies, such as CRISPR-Cas9, provide new hope for correcting the genetic mutations underlying HD. This review article aims to review the potential and effectiveness of CRISPR-Cas9 technology as a therapeutic tool for HD. The writing method used was Systematic Literature Review, with literature searches conducted using three databases: PubMed, ScienceDirect, and SpringerLink. This study reviewed several studies that used in vivo and in vitro models to evaluate the impact of HTT gene editing on mutant huntingtin protein expression and HD symptoms. Results showed that CRISPR-Cas9 can effectively reduce mutant huntingtin protein expression, reduce neuronal toxicity, and improve motor symptoms in mouse models of HD. Although these results are promising, further studies are needed to optimize the safety and effectiveness of using CRISPR-Cas9 in genetic therapy for HD.
Huntington’s Disease (HD) adalah kelainan neurodegeneratif yang disebabkan oleh ekspansi tripel CAG (>36) pada ekson pertama gen HTT yang mengkode protein huntingtin. Kemajuan teknologi pengeditan gen, seperti CRISPR-Cas9, memberikan harapan baru untuk mengoreksi mutasi genetik yang mendasari HD. Review artikel ini bertujuan untuk meninjau potensi dan efektivitas teknologi CRISPR-Cas9 sebagai alat terapi untuk HD. Metode penulisan yang digunakan adalah Systematic Literature Review, dengan pencarian studi literatur dilakukan menggunakan tiga basis data: PubMed, ScienceDirect, dan SpringerLink. Penelitian ini mengkaji beberapa studi yang menggunakan model in vivo dan in vitro untuk mengevaluasi dampak pengeditan gen HTT terhadap ekspresi protein huntingtin mutan dan gejala HD. Hasil menunjukkan bahwa CRISPR-Cas9 dapat secara efektif mengurangi ekspresi protein huntingtin mutan, mengurangi toksisitas neuronal, dan memperbaiki gejala motorik pada model tikus HD. Meskipun hasil ini menjanjikan, penelitian lebih lanjut diperlukan untuk mengoptimalkan keamanan dan efektivitas penggunaan CRISPR-Cas9 dalam terapi genetik untuk HD.
Title: Tinjauan Literatur Sistematis; Pemanfaatan Teknologi CRISPR-CAS9 Untuk Pengobatan Penyakit Huntington’s
Description:
A Systematic Literature Review: Utilization of CRISPR-CAS9 Technology for Treating Huntington's Disease
Huntington's Disease (HD) is a neurodegenerative disorder caused by a CAG triple expansion (>36) in the first exon of the HTT gene encoding the huntingtin protein.
Advances in gene editing technologies, such as CRISPR-Cas9, provide new hope for correcting the genetic mutations underlying HD.
This review article aims to review the potential and effectiveness of CRISPR-Cas9 technology as a therapeutic tool for HD.
The writing method used was Systematic Literature Review, with literature searches conducted using three databases: PubMed, ScienceDirect, and SpringerLink.
This study reviewed several studies that used in vivo and in vitro models to evaluate the impact of HTT gene editing on mutant huntingtin protein expression and HD symptoms.
Results showed that CRISPR-Cas9 can effectively reduce mutant huntingtin protein expression, reduce neuronal toxicity, and improve motor symptoms in mouse models of HD.
Although these results are promising, further studies are needed to optimize the safety and effectiveness of using CRISPR-Cas9 in genetic therapy for HD.
Huntington’s Disease (HD) adalah kelainan neurodegeneratif yang disebabkan oleh ekspansi tripel CAG (>36) pada ekson pertama gen HTT yang mengkode protein huntingtin.
Kemajuan teknologi pengeditan gen, seperti CRISPR-Cas9, memberikan harapan baru untuk mengoreksi mutasi genetik yang mendasari HD.
Review artikel ini bertujuan untuk meninjau potensi dan efektivitas teknologi CRISPR-Cas9 sebagai alat terapi untuk HD.
Metode penulisan yang digunakan adalah Systematic Literature Review, dengan pencarian studi literatur dilakukan menggunakan tiga basis data: PubMed, ScienceDirect, dan SpringerLink.
Penelitian ini mengkaji beberapa studi yang menggunakan model in vivo dan in vitro untuk mengevaluasi dampak pengeditan gen HTT terhadap ekspresi protein huntingtin mutan dan gejala HD.
Hasil menunjukkan bahwa CRISPR-Cas9 dapat secara efektif mengurangi ekspresi protein huntingtin mutan, mengurangi toksisitas neuronal, dan memperbaiki gejala motorik pada model tikus HD.
Meskipun hasil ini menjanjikan, penelitian lebih lanjut diperlukan untuk mengoptimalkan keamanan dan efektivitas penggunaan CRISPR-Cas9 dalam terapi genetik untuk HD.
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