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.

Genetic conditions amenable to ASO therapy.

Duchenne Muscular Dystrophy

Spinal Muscular Atrophy

Dravet Syndrome

Hereditary Transthyretin Amyloidosis

Huntington’s Disease

Amyotrophic Lateral Sclerosis (SOD1)

Angelman Syndrome

Usher Syndrome Type 2A

Leber Congenital Amaurosis Type 10

Myotonic Dystrophy Type 1

Rett Syndrome

Batten Disease (CLN3)

MELAS Syndrome

Friedreich Ataxia

Charcot-Marie-Tooth Disease Type 1A

Marfan Syndrome

Osteogenesis Imperfecta

Ehlers-Danlos Syndrome (Classical Type)

Prader-Willi Syndrome

Fragile X Syndrome

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