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Hurler syndrome UPSC NOTE

 Why in News

  • A treatment protocol that combines autologous stem cell transplants with gene therapy shows signs of correcting skeletal deformities in eight children with Hurler syndrome.

  • A rare disease that stunts skeletal system growth.

  • Promising results from a phase 1 trial suggest that similar strategies may counteract one of the debilitating complications of this rare disorder, which has no cure. 

  • Patients who received autologous stem cells containing the corrected gene displayed close-to-normal skeletal growth patterns.

Hurler syndrome

  • Hurler syndrome, also known as mucopolysaccharidosis type I (MPS I).

  • It is a rare genetic disorder

  • It's a lysosomal storage disease, which means the body is missing or has a deficiency in a specific enzyme needed to break down complex sugar molecules called glycosaminoglycans (GAGs).

What is stem cell

  • They can divide and copy themselves over a long period of time, replenishing their pool.

  • They have the potential to develop into many different types of specialized cells, like muscle cells, nerve cells, or blood cells.

  •  This characteristic is what makes them so valuable.

There are two main types of stem cells:

  • Embryonic stem cells (ESCs): These are pluripotent, meaning they have the ability to develop into any cell type in the human body. 

  • They are derived from the inner cell mass of a blastocyst, an early-stage embryo

  • Due to ethical considerations, research on embryonic stem cells is limited in many countries.

  • Adult stem cells (ASCs): These are found in various tissues throughout the body, like bone marrow, blood, or fat

  • They are multipotent, meaning they can develop into a limited number of cell types specific to their tissue of origin. 

  • They can be a valuable source of cells for regenerative medicine.



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Learnerz IAS | Concept oriented UPSC Classes in Malayalam: Hurler syndrome UPSC NOTE
Hurler syndrome UPSC NOTE
Learnerz IAS | Concept oriented UPSC Classes in Malayalam
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