Successful experiment of gene therapy for the treatment of ‘hemophilia’ developed by Pfizer

After the successful results of a gene therapy treatment trial developed by American pharmaceutical company ‘Pfizer’ to treat the hereditary blood disease ‘Hemophilia’, experts have expressed hope that the method will lead to quicker and cheaper treatment.

‘Hemophilia’ is an inherited blood disease that is usually passed on from the father or earlier ancestors to the new generation.

Usually, the disease does not affect women, but if a woman’s father is suffering from this disease, then the woman’s children can also be affected by it.

People with this disease lack a chemical called Factor IX, due to which they have blood clotting problems and their body starts bleeding.



So far, patients around the world have to get injections equipped with a chemical called Factor IX every second or third day, which strengthens their liver and temporarily relieves them from blood clotting.


People with this disease have to get injections throughout their lives and they also have to take care of its adherence, due to which they cannot live an independent life.


However, now the US company ‘Pfizer’ has claimed that the results of its therapy trial have been encouraging, raising hopes that the method will be cheaper and standard.


According to the news agency ‘Reuters’, the results of the third and important trial of ‘Gene Therapy’ by Pfizer showed that the patient only benefits from treatment once and his body starts making blood on its own.


Under the method of gene therapy, medicines are inserted into the patient’s body that work to improve the damaged or mutated genes.


Under this treatment, medicines are delivered to the patient’s body with the help of vectors( DNA Molecule) through injection to the place that is infected with the disease or virus.


The vector system plays the role of a vehicle or path in a way and delivers the drug inserted into the patient’s body to the place where the virus or disease is present.


According to a statement issued by Pfizer, under the above procedure, the patient needs treatment only once, after which the human body automatically starts producing a chemical called Factor IX in the blood.


Lack of a chemical called Factor IX causes hemophilia and starts bleeding from the human body, but under the new method of treatment, the chemical will start making on its own.


Until now, hemophilia patients have been given Factor ix chemicals through fusion injections or drips to protect them from bleeding.




4th Professional Medical Student. Karachi Medical and Dental College.

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