Genetic Conditions

Browse our database of genetic conditions with information from GeneReviews.

Lung Disorders AR

Cystic Fibrosis

Gene: CFTR

A life-threatening genetic disorder that affects the lungs and digestive system. Caused by mutations in the CFTR gene, it results in thick, sticky mucus that clogs airways and blocks the …

Treatment Available
Cancer Risk AD

Hereditary Breast and Ovarian Cancer (BRCA1/2)

Genes: BRCA1, BRCA2

An inherited cancer syndrome that significantly increases the risk of breast and ovarian cancers. BRCA1 carriers have up to 72% lifetime breast cancer risk.

Treatment Available
Cancer Risk AD

Lynch Syndrome

Genes: MLH1, MSH2, MSH6, PMS2, EPCAM

Hereditary colorectal cancer syndrome that increases risk of colorectal, endometrial, and other cancers. Lifetime colorectal cancer risk up to 80%.

Treatment Available
Cardiovascular AD

Familial Hypercholesterolemia

Genes: LDLR, APOB, PCSK9

Inherited high cholesterol that leads to early heart disease if untreated. LDL cholesterol is significantly elevated from birth.

Treatment Available
Blood Disorders AR

Sickle Cell Disease

Gene: HBB

A blood disorder causing abnormal hemoglobin that distorts red blood cells into a sickle shape, causing pain crises and organ damage.

Treatment Available
Neurological AD

Huntington Disease

Gene: HTT

A progressive brain disorder causing uncontrolled movements, emotional problems, and loss of thinking ability. Symptoms typically begin in adulthood.

Supportive Care
Neurological AR

Spinal Muscular Atrophy

Gene: SMN1

A genetic neuromuscular disease affecting motor neurons, causing progressive muscle weakness. New treatments have transformed outcomes.

Treatment Available
Metabolic AR

Phenylketonuria (PKU)

Gene: PAH

A metabolic disorder preventing breakdown of phenylalanine. Without dietary treatment, causes intellectual disability. Highly treatable with early diagnosis.

Treatment Available
Neurological AR

Tay-Sachs Disease

Gene: HEXA

A fatal genetic disorder causing progressive destruction of nerve cells. Classic infantile form causes death by age 4-5.

Supportive Care
Developmental XL

Fragile X Syndrome

Gene: FMR1

The most common inherited cause of intellectual disability and autism spectrum disorder. More severe in males.

Supportive Care