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PTC258 is a specific and orally active splicing modulator of Elongator complex protein 1 gene (ELP1). PTC258 increases the expression of ELP1 in vitro and in vivo. PTC258 is well tolerated in mouse model.

For research use only. We do not sell to patients.

PTC258 Chemical Structure

PTC258 Chemical Structure

CAS No. : 2476724-74-8

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1 mg USD 360 In-stock
5 mg USD 1080 In-stock
10 mg USD 1750 In-stock
50 mg USD 4200 In-stock
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Based on 1 publication(s) in Google Scholar

PTC258 Related Antibodies

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Description

PTC258 is a specific and orally active splicing modulator of Elongator complex protein 1 gene (ELP1). PTC258 increases the expression of ELP1 in vitro and in vivo. PTC258 is well tolerated in mouse model[1].

In Vitro

PTC258 (0.01 nM-0.01 μM; 48 h) efficiently increases full-length ELP1 mRNA and protein in Familial Dysautonomia (FD) patient fibroblasts[1].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

PTC258 Related Antibodies
In Vivo

PTC258 (3-24 mg/kg; p.o.; once daily for 3 months) is well tolerated. It corrects the splicing of the ELP1 transcript and significantly increases the amount of functional protein in vivo in all tissues tested, including the brain in mouse model[1].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Animal Model: Familial Dysautonomia (FD) Mouse model[1]
Dosage: 3 mg/kg, 6 mg/kg, 12 mg/kg, 24 mg/kg
Administration: Oral gavage; once daily for 3 month
Result: Increased full-length ELP1 transcript in a dose-dependent manner. And also, it increased in functional ELP1 protein in the brain, trigeminal, liver, and quadricep.
Molecular Weight

351.92

Formula

C16H18ClN3S2
CAS No.
Appearance

Solid

Color

White to yellow

SMILES

CC(C1=NC(Cl)=CC(NCC2=CC=CS2)=C1S3)=C3C[C@H](C)N
Shipping

Room temperature in continental US; may vary elsewhere.
Storage

-20°C, protect from light, stored under nitrogen

*In solvent : -80°C, 6 months; -20°C, 1 month (protect from light, stored under nitrogen)

Solvent & Solubility
In Vitro: 

DMSO : 100 mg/mL (284.16 mM; Need ultrasonic; Hygroscopic DMSO has a significant impact on the solubility of product, please use newly opened DMSO)

Preparing
Stock Solutions
Concentration Solvent Mass 1 mg 5 mg 10 mg
1 mM 2.8416 mL 14.2078 mL 28.4155 mL
5 mM 0.5683 mL 2.8416 mL 5.6831 mL
View the Complete Stock Solution Preparation Table

* Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (protect from light, stored under nitrogen). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.

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Mass (g) = Concentration (mol/L) × Volume (L) × Molecular Weight (g/mol)

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Concentration (start) × Volume (start) = Concentration (final) × Volume (final)

This equation is commonly abbreviated as: C1V1 = C2V2

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Working solution concentration: mg/mL
Purity & Documentation
References

Complete Stock Solution Preparation Table

* Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (protect from light, stored under nitrogen). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.

Optional Solvent Concentration Solvent Mass 1 mg 5 mg 10 mg 25 mg
DMSO 1 mM 2.8416 mL 14.2078 mL 28.4155 mL 71.0389 mL
5 mM 0.5683 mL 2.8416 mL 5.6831 mL 14.2078 mL
10 mM 0.2842 mL 1.4208 mL 2.8416 mL 7.1039 mL
15 mM 0.1894 mL 0.9472 mL 1.8944 mL 4.7359 mL
20 mM 0.1421 mL 0.7104 mL 1.4208 mL 3.5519 mL
25 mM 0.1137 mL 0.5683 mL 1.1366 mL 2.8416 mL
30 mM 0.0947 mL 0.4736 mL 0.9472 mL 2.3680 mL
40 mM 0.0710 mL 0.3552 mL 0.7104 mL 1.7760 mL
50 mM 0.0568 mL 0.2842 mL 0.5683 mL 1.4208 mL
60 mM 0.0474 mL 0.2368 mL 0.4736 mL 1.1840 mL
80 mM 0.0355 mL 0.1776 mL 0.3552 mL 0.8880 mL
100 mM 0.0284 mL 0.1421 mL 0.2842 mL 0.7104 mL
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PTC258 Related Classifications

Help & FAQs
  • Do most proteins show cross-species activity?

    Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

Keywords:

PTC2582476724-74-8PTC 258PTC-258OthersInhibitorinhibitorinhibit

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