Genetic Diseases

Genetic Testing for Phenylketonuria (PKU)

PKU results from PAH deficiency leading to phenylalanine accumulation and brain damage.

Genetic Diseases

What is PKU?

An amino acid metabolic disorder.

Symptoms

Growth delay and intellectual disability without early treatment.

Genetic causes

PAH gene mutations.

Inheritance pattern

Autosomal recessive.

Important genes

PAH.

Who should be tested?

Neonates via screening and affected families.

Recommended tests

PAH sequencing and carrier screening.

Counseling

PGT-M for carrier couples.

Frequently Asked Questions

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Gene Negar Ayandegan · Genetic Testing for Phenylketonuria (PKU)