Genetic Substrate Reduction Therapy: A Promising Approach for Lysosomal Storage Disorders
Abstract
:1. Introduction
2. RNA Interference (RNAi) as a Mechanism to Promote Substrate Reduction
2.1. RNAi-Mediated SRT for Gaucher Disease
2.2. RNAi-Mediated SRT for Mucopolysaccharidoses
3. Antisense Oligonucleotides (AOs) as Tools to Achieve Substrate Reduction
Antisense Oligonucleotide-Mediated SRT for Pompe Disease
4. Conclusions
Acknowledgments
Author Contributions
Conflicts of Interest
References
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Drug | Target | Condition | Phase | Status |
---|---|---|---|---|
RNAi | ||||
DCR-MYC | Myc | Solid tumors Hepatocellular carcinoma | I | Recruiting |
I/II | Recruiting | |||
ALN-TTRSC (Revusiran) | Transthyretin | TTR-mediated familial amyloidotic cardiomyopathy | I | Completed |
II | Active | |||
III | Active | |||
ALN-CC5 | Complement component C5 | Paroxysmal nocturnal Hemoglobinuria | I/II | Active |
ALN-AS1 | ALAS-1 | Acute intermittent porphyria | I | Recruiting |
ALN-PCSSC | PCSK9 | Hypercholesterolemia | I | Completed |
ALN-TTR02 (Patisiran) | Transthyretin | TTR-mediated amyloidosis | II | Active |
III | Active | |||
ALN-AT3SC | Antithrombin | Hemophilia A/B | I | Recruiting |
TKM-080301 | Polo-like kinase 1 | Hepatocellular carcinoma Neuroendocrine tumors | I | Active |
I/II | Recruiting | |||
TKM-100802 | Ebola genome | Ebola virus | I/II | Terminated |
QPI-1007 | Caspase 2 | Primary angle-closure glaucoma | II | Completed |
RXI-109 | CTGF | Hypertrophic scar keloid excision surgery | II | Completed |
II | Completed | |||
Atu027 | PKN3 | Pancreatic ductal carcinoma | I/II | Completed |
SYL040012 | Β2-adrenergic receptor | Ocular hypertension | II | Completed |
Antisense | ||||
Mipomersen * | ApoB 100 | Heterozygous familial hypercholesterolemia atherosclerosis | III | Completed |
III | Completed | |||
ISIS-TTRRX | Transthyretin | Familial amyloid polyneuropathy | III | Active |
ISIS-ApoC-IIIRX | ApoCIII | Hypertriglyceridemia | II | Completed |
ISIS-DMPKRX | DMPK | Myotonic dystrophy type 1 | I/II | Recruiting |
ISIS APO(a)-LRX | Apoliprotein (a) | Elevated lipoprotein (a) | I | Completed |
II | Completed | |||
Curtirsen | Clusterin | Non-small cell lung cancer Prostate cancer | III | Recruiting |
III | Active |
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Coutinho, M.F.; Santos, J.I.; Matos, L.; Alves, S. Genetic Substrate Reduction Therapy: A Promising Approach for Lysosomal Storage Disorders. Diseases 2016, 4, 33. https://doi.org/10.3390/diseases4040033
Coutinho MF, Santos JI, Matos L, Alves S. Genetic Substrate Reduction Therapy: A Promising Approach for Lysosomal Storage Disorders. Diseases. 2016; 4(4):33. https://doi.org/10.3390/diseases4040033
Chicago/Turabian StyleCoutinho, Maria Francisca, Juliana Inês Santos, Liliana Matos, and Sandra Alves. 2016. "Genetic Substrate Reduction Therapy: A Promising Approach for Lysosomal Storage Disorders" Diseases 4, no. 4: 33. https://doi.org/10.3390/diseases4040033