genomics-pipeline

Step 9: Short Tandem Repeat (STR) Expansion Screening

What This Does

Screens for pathogenic repeat expansions — a class of mutations invisible to both DeepVariant and Manta. In these diseases, a short DNA sequence (3-6 bases) gets repeated too many times.

Why

STR expansions cause ~40 known neurological/neuromuscular diseases including Huntington’s, Fragile X, Friedreich’s ataxia, ALS/FTD, myotonic dystrophy, and multiple spinocerebellar ataxias.

Tool

Docker Image

weisburd/expansionhunter:latest

Key Disease Thresholds

| Disease | Gene | Repeat Unit | Normal | Pathogenic | |—|—|—|—|—| | Huntington’s | HTT | CAG | <27 | >35 | | Fragile X | FMR1 | CGG | <45 | ≥55 (premutation) / >200 (full) | | Friedreich’s Ataxia | FXN | GAA | <33 | >66 | | ALS/FTD | C9ORF72 | GGCCCC | <24 | >30 | | Myotonic Dystrophy 1 | DMPK | CTG | <35 | >50 | | SCA1 | ATXN1 | CAG | <33 | >39 | | SCA2 | ATXN2 | CAG | <22 | >33 |

FMR1 Clinical Zones

FMR1 (Fragile X) has four distinct clinical zones — the intermediate zone (45-54 repeats) is often omitted but clinically relevant:

Zone Repeats Clinical Significance
Normal <45 No risk
Intermediate (gray zone) 45-54 Not affected, but repeats may expand in offspring. Genetic counseling recommended for carriers.
Premutation 55-200 Risk of FXTAS (tremor/ataxia, males >50), FXPOI (premature ovarian insufficiency). Offspring at risk of full expansion.
Full mutation >200 Fragile X syndrome (intellectual disability, behavioral features). Penetrance varies by sex and methylation.

Notes