Improvements In Health - Gene Therapy

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Everyday, there are new innovations and improvements in the science and health sector and one of such innovations that remains a debate till date for a number of reasons is gene therapy or gene manipultion. I'm certain everyone of us has our own thoughts on the topic and no matter which side of the debate you choose, there is no denying gene therapy has its benefits.

One of these improvements I would like to talk a bit about is in sickle cell diseaae. The sickle cell disease as we know is a very serious hereditary disease that has had one cure till about a few years ago when 2 other therapies were approved. These therapies are 2 gene therapies that were approved by the are Casgevy and Lyfgenia.

_{Shape of sickle cell}

Casgevy is a gene replacement therapy that is used only on sickle cell patients 12 years and above. What is known as CRISPR or Cas 9 is used to cut DNA in targetted areas. These cut areas are then edited or replaced with modified versions of the cut DNA which then attach and multiply within the bone marrow of the patient. In this method of curing sickle cell, what we are generally doing is dding the fetal heamoglobin which we normally see in new borns not older than the age of 1. This fetal heamoglobin prevents the sickle cell from activating and so as the modified cut DNA is introduced into the sickle cell patient, he or she is cured.

_{red blood cell}

The second approved gene therapy method is the Lyfgenia is cell based. It is also applied to patiets 12 years and above with the lentiviral vector. The sickle cell's patient’s blood stem cells is modified to produce a derived haemoglobin that works like haemoglobin A. This haemoglobin is seen in people without sickle cell and so modifying the patient's genes to have this haemophilia A will help them to not exhibit sickle cell disease.

These are not a 100% guarantee of working for everyone but has proven to work for more than 90% of the test sample in Casgevy and more than 80% in Lyfgenia.

So, these are just a summary of what these 2 gene therapies can do and how they are helping to cure sickle cell. To read more about these, visit Yalemedicine.org

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Thank you.