Background
Per virtutem suae unique technica characteres, Sirna medicamento facta est focus in operam in agro novis medicamento investigationis et progressionem in annis, et est unus de maxime cursim developing gene therapies ad praesens. Compared with traditional small molecule drugs and antibody drugs, siRNA Drug can intervene from the source, with the advantages of fast target screening, high therapeutic efficiency, not easy to produce drug resistance, long-lasting effect, low toxicity, strong specificity, R & D success rate and so on, which is regarded as bringing the “in addition to small molecule drugs and antibody drugs” outside of the third wave of Pharmaceuticals. Tertium fluctus de pharmaceuticals praeter parvum molecule medicinae et antibody medicinae.
Tamen, ut novum genus de medicamento moleculis, Sirna medicinae sunt Suspendisse: et require eget modifications et medicamento partus systems ad amplio medicamento - formatariam parva moleculis et antibody medicamentis, quod opposuit novum provocare et antibody, quod opposuit, quod est in primis progressionem, quod est in provocare in mane progressionem in Medicamenta in primis.
Introductio de Sirna Medicamenta
Sirna medicinae, etiam notum sicut oligonucleotides (ADS), ad medicamento - Promptus Oligonucleootides typice XVIII - XXX NT in longitudinem. Parvus RNA Medicamenta includit parva intercedente RNA (Sirna), Antisense Oligonucleotide (ASO), Microna (Mirna), parva activum RNA (Sarna), Messenger RNA (mRNA), aptamer, et antibody (mRNA (mRNA (Sarna), Messenger RNA (mRNA), aptamer et antibody (mRNA), aptamer, et antibody - Oligonucleic Conjugate (Aoc).
Current Research Focuses in tres genera Sirna Medicamenta: ASO, Sirna et Aptamer. Diversis oligonucleootides habent diversas machinationes actionis, et ludere partes inhibitors ad diversas gradus Pathogenesis. ASO SIRNA agit planum target mRNA; APAMER directe inhibits operatio de proteins involved in Pathogenesis. Quorum communis punctum est quod interveniendi in expressio target genes ad consequi ad morbus curatio.
Altiore, unicum mechanism actio facit parva nucleic acidum medicamenta multa commoda: princeps therapeuticam efficientiam, fortis target specifica, et levare, longecule medicamentis et medicamento resistentia, longa et inquisitionis, et alta res resistentia, et in parva acidum medicamina et medicamento resistentia, longa et admodum ad medicamento resistentia, et in parva acidum medicinae et medicamento resistentia, longa et inquisitionem, et alta res resistentiam Et progressionem, quae est in tertia generatione PHARMACEUTICICUS innovation technology quod promoveam mutationem de pharmaceutical industria sequentibus parva molecule medicinae et antibody medicinae. More et PHARMACEUTICAL societates habere invested in in eget innovative investigationis et progressionem Sirna medicinae.
Status parva nucleic acidum medicinae in foro
Probatus parva nucleic acidum medicamina:
- Antisense Oligonucleleotides (Asos) Hi una - subductis DNA aut RNA moleculis ligare ad specifica mRNA sequences ad inhibere translatione vel modificare splicing. Notabile Asos includit:
- Nusinersen (Spinraza): probatus pro spinae musculorum atrophia.
- Etpepirsens (Exondys LI): peltas Duchenne musculorum dystrophy.
- Inotersen (Tegsedi): De hereditario transthyretin - Mediated amyloidosis.
- PRINCIPIUM PRINCIPIUM RN (Sirnas): Geminus - RNA Molecules quod promovere degradationem target mRNA.
Probatus Sirna therapies includit:
- Patisiran (Onpattro): Primum FDA - probatus Sirna medicamento, addressing hereditario transthyretin - mediated amyloidosis.
- Givosiran (Givlaari), quia acuti hepatic porphyria.
- Lumasiran (Oxlumo) Targets Primary Hyperoxaluria Type I.
- Aptapers: Short, structured nucleic acida qui ligare ad specifica proteins.
- Pegaptanib (Macugen): An APAMER probatus pro Age - Maculation Degeneratum.
Keywords: Galnac - Sirna, Sirna Delivery, Sirna Effugere, iecoris lysosomes, Hepatocyte Lysosomes, tritosome, lysosome catabolism, Lysosomal stabilitatem, lysosomal acidum phosphatase
Post tempus: MMXXV - IV - IX 10:04:06