RNA·올리고 치료
성숙
siRNA·안티센스(ASO)로 특정 유전자의 발현 자체를 끄거나 조절하는 핵산 치료.
전망 희귀·대사질환에서 자리잡음. 간 밖 조직으로의 전달과 장기 지속형이 다음 과제.
개발 기업
최신 연구
전체 →Current opinion in microbiology
안티센스 올리고뉴클레오티드: 미생물학에서의 설계, 구현 및 향후 전망bioRxiv (프리프린트)
Nanopore-based profiling of PEGylation in nucleic acid therapeuticsbioRxiv (프리프린트)
Genome-wide functional genomics screens identify therapeutic vulnerabilities for lymphangioleiomyomatosisProceedings of the National Academy of Sciences of the United States of America
IVT-free, chemically synthesized protein-encoding RNA oligonucleotides for rapid production of personalized cancer vaccinesThe Journal of clinical endocrinology and metabolism
Approach to the Patient with Elevated Lipoprotein(a)Nature biomedical engineering
RModBlock antisense oligonucleotides as a universal tool for precise and efficient inhibition of RNA modificationsEpilepsy & behavior : E&B
From synaptic dysfunction to targeted intervention: a comprehensive review of STXBP1 encephalopathy and precision therapeutic strategiesThe Journal of clinical investigation
The splice of life: an isoform-centric view of disease, technology, and therapeuticsNature
An ATPγS recycling strategy for practical biocatalytic thiophosphorylationMolecular psychiatry
Translatable electrophysiological and behavioral abnormalities in a humanized model of SYNGAP1-disorderJCI insight
PPMO therapy for dysferlinopathy induces pseudoexon skipping and restoration of functional proteinProceedings of the National Academy of Sciences of the United States of America
Oncogenic snoRNA SNORD78 fuels colorectal cancer by protecting the m(6)A reader IMP2 to enhance phospholipid metabolism
진행 중 임상
전체 →- 1/2상모집 중A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor
- 단계 미표기모집 중Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis
- 3상모집 중A Study to Evaluate the Efficacy and Safety of Sefaxersen (RO7434656) in Participants With Primary Immunoglobulin A (IgA) Nephropathy at High Risk of Progression
- 2상모집 중단 (진행 중)Long-term Follow-up Study to Evaluate Durability of Treatment Response in Previous Bepirovirsen Study Participants (B-Sure)
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