A review on Gaucher disease: therapeutic potential of β-glucocerebrosidase-targeted mRNA/saRNA approach

Feng, Shunping; Rcheulishvili, Nino; Jiang, Xiaoming; Zhu, Pan; Pan, Xuehua; Wei, Meilan; Wang, Peng George; Ji, Yang; Papukashvili, Dimitri

doi:10.7150/ijbs.87741

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Int J Biol Sci 2024; 20(6):2111-2129. doi:10.7150/ijbs.87741 This issue Cite

Review

A review on Gaucher disease: therapeutic potential of β-glucocerebrosidase-targeted mRNA/saRNA approach

Shunping Feng^1,#, Nino Rcheulishvili^1,2,#, Xiaoming Jiang³, Pan Zhu³, Xuehua Pan⁴, Meilan Wei¹, Peng George Wang^1,✉, Yang Ji^1,✉, Dimitri Papukashvili^1,2,✉

1. Department of Pharmacology, School of Medicine, Southern University of Science and Technology, Shenzhen 518000, China.
2. Institute of Microbiology, Chinese Academy of Sciences, Beijing 100101, China.
3. Cheerland Biomedicine, Shenzhen, China.
4. Shenzhen Pengbo Biotech Co. Ltd, Shenzhen, China.
^# Contributed equally.

Citation:

Feng S, Rcheulishvili N, Jiang X, Zhu P, Pan X, Wei M, Wang PG, Ji Y, Papukashvili D. A review on Gaucher disease: therapeutic potential of β-glucocerebrosidase-targeted mRNA/saRNA approach. Int J Biol Sci 2024; 20(6):2111-2129. doi:10.7150/ijbs.87741. https://www.ijbs.com/v20p2111.htm

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Abstract

Gaucher disease (GD), a rare hereditary lysosomal storage disorder, occurs due to a deficiency in the enzyme β-glucocerebrosidase (GCase). This deficiency leads to the buildup of substrate glucosylceramide (GlcCer) in macrophages, eventually resulting in various complications. Among its three types, GD2 is particularly severe with neurological involvements. Current treatments, such as enzyme replacement therapy (ERT), are not effective for GD2 and GD3 due to their inability to cross the blood-brain barrier (BBB). Other treatment approaches, such as gene or chaperone therapies are still in experimental stages. Additionally, GD treatments are costly and can have certain side effects. The successful use of messenger RNA (mRNA)-based vaccines for COVID-19 in 2020 has sparked interest in nucleic acid-based therapies. Remarkably, mRNA technology also offers a novel approach for protein replacement purposes. Additionally, self-amplifying RNA (saRNA) technology shows promise, potentially producing more protein at lower doses. This review aims to explore the potential of a cost-effective mRNA/saRNA-based approach for GD therapy. The use of GCase-mRNA/saRNA as a protein replacement therapy could offer a new and promising direction for improving the quality of life and extending the lifespan of individuals with GD.

Keywords: Gaucher disease, mRNA, saRNA, GlcCer, GCase, lysosomal storage disease, ERT, SRT, protein replacement therapy

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APA

Feng, S., Rcheulishvili, N., Jiang, X., Zhu, P., Pan, X., Wei, M., Wang, P.G., Ji, Y., Papukashvili, D. (2024). A review on Gaucher disease: therapeutic potential of β-glucocerebrosidase-targeted mRNA/saRNA approach. International Journal of Biological Sciences, 20(6), 2111-2129. https://doi.org/10.7150/ijbs.87741.

ACS

Feng, S.; Rcheulishvili, N.; Jiang, X.; Zhu, P.; Pan, X.; Wei, M.; Wang, P.G.; Ji, Y.; Papukashvili, D. A review on Gaucher disease: therapeutic potential of β-glucocerebrosidase-targeted mRNA/saRNA approach. Int. J. Biol. Sci. 2024, 20 (6), 2111-2129. DOI: 10.7150/ijbs.87741.

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CSE

Feng S, Rcheulishvili N, Jiang X, Zhu P, Pan X, Wei M, Wang PG, Ji Y, Papukashvili D. 2024. A review on Gaucher disease: therapeutic potential of β-glucocerebrosidase-targeted mRNA/saRNA approach. Int J Biol Sci. 20(6):2111-2129.

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