WARNING: This product is for research use only, not for human or veterinary use.
Hodoodo CAT#: H130246
CAS#: 2476724-74-8
Description: PTC258 is an agent that passes the blood brain barrier and leads to the correct splicing of ELP1 for the treatment of familial dysautonomia in mice.
Hodoodo Cat#: H130246
Name: PTC258
CAS#: 2476724-74-8
Chemical Formula: C16H18ClN3S2
Exact Mass: 351.06
Molecular Weight: 351.910
Elemental Analysis: C, 54.61; H, 5.16; Cl, 10.07; N, 11.94; S, 18.22
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Synonym: PTC258; PTC 258; PTC-258
IUPAC/Chemical Name: (S)-2-(2-aminopropyl)-5-chloro-3-methyl-N-(thiophen-2-ylmethyl)thieno[3,2-b]pyridin-7-amine
InChi Key: XBAVUBLRECEOPY-VIFPVBQESA-N
InChi Code: InChI=1S/C16H18ClN3S2/c1-9(18)6-13-10(2)15-16(22-13)12(7-14(17)20-15)19-8-11-4-3-5-21-11/h3-5,7,9H,6,8,18H2,1-2H3,(H,19,20)/t9-/m0/s1
SMILES Code: CC1=C(C[C@@H](N)C)SC2=C(NCC3=CC=CS3)C=C(Cl)N=C21
Appearance: To be determined
Purity: >98% (or refer to the Certificate of Analysis)
Shipping Condition: Shipped under ambient temperature as non-hazardous chemical. This product is stable enough for a few weeks during ordinary shipping and time spent in Customs.
Storage Condition: Dry, dark and at 0 - 4 C for short term (days to weeks) or -20 C for long term (months to years).
Solubility: To be determined
Shelf Life: >2 years if stored properly
Drug Formulation: To be determined
Stock Solution Storage: 0 - 4 C for short term (days to weeks), or -20 C for long term (months).
HS Tariff Code: 2934.99.9001
More Info:
Biological target: | |
In vitro activity: | |
In vivo activity: |
The following data is based on the product molecular weight 351.91 Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.
Concentration / Solvent Volume / Mass | 1 mg | 5 mg | 10 mg |
---|---|---|---|
1 mM | 1.15 mL | 5.76 mL | 11.51 mL |
5 mM | 0.23 mL | 1.15 mL | 2.3 mL |
10 mM | 0.12 mL | 0.58 mL | 1.15 mL |
50 mM | 0.02 mL | 0.12 mL | 0.23 mL |
Formulation protocol: | |
In vitro protocol: | |
In vivo protocol: |
1. Morini E, Chekuri A, Logan EM, Bolduc JM, Kirchner EG, Salani M, Krauson AJ, Narasimhan J, Gabbeta V, Grover S, Dakka A, Mollin A, Jung SP, Zhao X, Zhang N, Zhang S, Arnold M, Woll MG, Naryshkin NA, Weetall M, Slaugenhaupt SA. Development of an oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia. Am J Hum Genet. 2023 Feb 15:S0002-9297(23)00044-7. doi: 10.1016/j.ajhg.2023.01.019. Epub ahead of print. PMID: 36809767.