63865-89-4

Upstream product

• 1070-19-5 N-(tert-butyloxycarbonyl) azide 
• 5123-49-9 N⁵-trifluoroacetyl-L-ornithine 
• 383-63-1 ethyl trifluoroacetate, 
• 21887-64-9 N₂-(tert-butoxycarbonyl)-L-ornithine 
• 2480-93-5 Boc-Orn(Z)-OH 

Downstream Products

• 376648-83-8 benzyl (2S)-2-[(2S)-2-[(tert-butoxycarbonyl)amino]-5-(2,2,2-trifluoroacetamido)pentanamido]-5-(2,2,2-trifluoroacetamido)pentanoate 
• 376648-82-7 benzyl (2S)-2-[(tert-butoxycarbonyl)amino]-5-(2,2,2-trifluoroacetamido)pentanoate 
• 376648-86-1 2-[2-[2-[2-tert-butoxycarbonylamino-5-(2,2,2-trifluoro-acetylamino)-pentanoylamino]-5-(2,2,2-trifluoro-acetylamino)-pentanoylamino]-5-(2,2,2-trifluoro-acetylamino)-pentanoylamino]-5-(2,2,2-trifluoro-acetylamino)-pentanoic acid benzyl ester 

Relevant academic research and scientific papers

METHODS AND COMPOUNDS FOR THE TREATMENT OF GENETIC DISEASE

The present disclosure relates to compounds and methods for modulating the expression of dmpk, and treating diseases and conditions in which dmpk plays an active role. The compound can be a transcription modulator molecule having a first terminus, a second terminus, and oligomeric backbone, wherein: a) the first terminus comprises a DNA-binding moiety capable of noncovalently binding to a nucleotide repeat sequence CAG or CTG; b) the second terminus comprises a protein-binding moiety binding to a regulatory molecule that modulates an expression of a gene comprising the nucleotide repeat sequence CAG or CTG; and c) the oligomeric backbone comprising a linker between the first terminus and the second terminus.

An efficient, scalable synthesis of the molecular transporter octaarginine via a segment doubling strategy.

[reaction: see text]. Short oligomers of arginine function as remarkably efficient molecular transporters of drugs and probe molecules into cells and tissue. Currently, these compounds are prepared on resin through a unidirectional solid-phase synthesis.