Gene Therapy Solutions

Genetic Disorders

Genetic disorders affect millions of people world-wide. Scientists have currently identified more than 4000 different genetic disorders.

There are four main types of genetic disorders. These include:

  • single-gene
  • multifactorial
  • chromosomal
  • mitochondrial

Single-gene disorders are caused by a defect in a single gene. Examples include Huntington's disease, cystic fibrosis, and sickle cell anemia. Multifactorial disorders are caused by a combination of genes. Alzheimer's, heart disease and even cancer can be influenced by multifactorial disorders. Chromosomal disorders, such as Down syndrome, are caused by changes or replications of an entire chromosome. Finally, there are mitochondrial disorders in which the DNA of mitochondria, tiny organelles used in cell metabolism, become affected.

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