Open AccessEffectiveness of Enzyme Replacement and Enhancement Therapies on the Management of Tay-Sachs
Author(s) -
Peterchris Okpala,
Sandra Okpala
Publication year - 2021
Publication title -
genetics and molecular medicine
Language(s) - English
Resource type - Journals
ISSN - 2689-1077
DOI - 10.33425/2689-1077.1009
Subject(s) - enzyme replacement therapy , medicine , disease , pediatrics , substrate reduction therapy , blindness , optometry
Tay-Sachs is a hereditary syndrome that damages the spinal cord and brain nerve cells. This rare type of disease is mainly manifested in infants at the ages of three to six months. Affected infants lose all their motor skills, such as sitting and crawling. As the disease evolves, kids infected will experience seizures, blindness, deafness, and eventually paralysis. Solovyeva et al., 2018). Currently, there is no treatment for TSD, and different therapies are joined to improve symptoms in patients with the late-onset form. Infant TSD patients do not survive beyond five years. These therapies can also defer the progression of TSD in late-onset form patients. Enzyme replacement therapy (ERT) and enzyme enhancement therapies (EET) are encouraging therapies that could finally cure the disease. Smith (2021) demonstrated the efficiency of ERT and EET therapies using α and β subunits of Hex A using rats and a rare form of sheep known as Jacob sheep. This evaluation discourses the effectiveness of Enzyme replacement and Enhancement therapies in preventing neuroinflammation and non-neurologic symptoms of the TSD.