RNAi therapeutics for diseases involving protein aggregation: fazirsiran for alpha-1 antitrypsin deficiency-associated liver disease.
Expert Opin Investig Drugs
; 32(7): 571-581, 2023.
Article
em En
| MEDLINE
| ID: mdl-37470509
When certain proteins in the body are not produced correctly, it can result in disease. Examples of such diseases include alpha-1 antitrypsin deficiency (AATD), hereditary transthyretin amyloidosis (ATTR), amyotrophic lateral sclerosis (ALS), Alzheimer's disease, and Parkinson's disease, where misfolded proteins can form harmful aggregates. Research is underway to develop new therapies such as small interfering RNA (siRNA), antisense oligonucleotides, monoclonal antibodies, clustered regularly interspaced short palindromic repeats (CRISPR), and small molecule protein modulators to prevent protein aggregates. An example of an experimental siRNA therapy for patients with AATD is fazirsiran, designed to prevent the production of mutated alpha-1 antitrypsin (Z-AAT) and allows the liver to clear previously accumulated Z-AAT protein, which is believed to be responsible for causing AATD liver disease. This review summarizes the development of fazirsiran and describes how it helps our understanding and treatment of diseases associated with protein aggregation.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Deficiência de alfa 1-Antitripsina
/
Agregados Proteicos
Tipo de estudo:
Risk_factors_studies
Limite:
Humans
Idioma:
En
Revista:
Expert Opin Investig Drugs
Assunto da revista:
TERAPIA POR MEDICAMENTOS
Ano de publicação:
2023
Tipo de documento:
Article
País de afiliação:
Alemanha