Question 1

What is a personalized ASO?

Accepted Answer

A personalized antisense oligonucleotide (ASO) is a short synthetic DNA strand designed specifically for one patient's unique genetic mutation. It binds complementary RNA to modulate gene expression — silencing a toxic gene, modifying splicing (exon-inclusion, exon-skipping, or pseudoexon-blocking), or upregulating an insufficient gene — without editing the genome.

Question 2

How do I get an ASO designed for my mutation?

Accepted Answer

Visit pequliar.ai and describe your genetic mutation or upload your genetic report. Pequliar's AI designs ranked, synthesis-ready ASO candidates with full chemistry assignment and vendor-formatted sequences. A complete design costs $399 and takes minutes.

Question 3

What diseases can be treated with ASOs?

Accepted Answer

ASOs can target nearly any genetic mutation. FDA-approved ASO drugs exist for spinal muscular atrophy (Spinraza — exon-inclusion), Duchenne muscular dystrophy (Exondys 51 — exon-skipping), ALS (Qalsody — gene-silencing), and hereditary transthyretin amyloidosis (Tegsedi — gene-silencing). Personalized ASOs have been designed for Dravet syndrome, MELAS, Usher syndrome, Angelman syndrome, Huntington's disease, Leber congenital amaurosis, and many other rare genetic diseases.

Question 4

How much does personalized ASO design cost?

Accepted Answer

A complete ASO design on Pequliar costs $399. This includes ranked candidates, full chemistry assignment, thermodynamic scoring, quality checks, and vendor-formatted sequences ready for ordering from synthesis vendors.

Question 5

Has Pequliar been validated?

Accepted Answer

In blind validation, Pequliar recovered the exact sequences behind FDA-approved ASO drugs — including Nusinersen (Spinraza), Tofersen (Qalsody), and Eteplirsen (Exondys 51) — that took years to develop, in minutes.

Notes on ASO design

ASO Design Tools: What Patients and Researchers Should Know