Introduction
Two children of Gautam Dongri in India and a son of Basakazi Mazizi in Tanzania live with a hereditary blood disorder that turns blood cells into machines of pain. Now that new gene therapies promise to cure their sickle cell disease, Dongri says, “I pray for the treatment to come to us.” But experts say the one-time treatment is not available in India and Africa – places where the disease is highly prevalent. Numerous vast barriers cut off the world from gene therapy in general.
The Struggle for Basic Treatment
For gene therapy to be an option, people in developing countries need to stay alive long enough to receive it. There, sickle cell disease exposes patients to disability or death more than in wealthy areas. Late diagnoses are common, and primary care can be difficult to access.
Major Challenges
These stark realities make the cost of gene therapies an insurmountable barrier, according to experts. The cost of gene therapies for sickle cell disease in the United States is $3.1 million and $2.2 million, although the cost of gene therapies can vary from country to country. The process of administering the treatments is a significant hurdle as well. Patients must go to hospitals, where stem cells are removed from their blood in a process that requires specialized equipment. One treatment requires sending cells to a lab as quickly as possible to keep them fresh and utilizing a gene-editing tool known as CRISPR to remove a gene. The cells must be sent back in liquid nitrogen to keep them frozen until they are needed. Patients must undergo chemotherapy before receiving their modified cells via IV, spending weeks in the hospital. The process can take several months.
Major Challenges
These stark realities make the cost of gene therapies an insurmountable barrier, according to experts. The cost of gene therapies for sickle cell disease in the United States is $3.1 million and $2.2 million, although the cost of gene therapies can vary from country to country. The process of administering the treatments is a significant hurdle as well. Patients must go to hospitals, where stem cells are removed from their blood in a process that requires specialized equipment. One treatment requires sending cells to a lab as quickly as possible to keep them fresh and utilizing a gene-editing tool known as CRISPR to remove a gene. The cells must be sent back in liquid nitrogen to keep them frozen until they are needed. Patients must undergo chemotherapy before receiving their modified cells via IV, spending weeks in the hospital. The process can take several months.
Major Challenges
These stark realities make the cost of gene therapies an insurmountable barrier, according to experts. The cost of gene therapies for sickle cell disease in the United States is $3.1 million and $2.2 million, although the cost of gene therapies can vary from country to country. The process of administering the treatments is a significant hurdle as well. Patients must go to hospitals, where stem cells are removed from their blood in a process that requires specialized equipment. One treatment requires sending cells to a lab as quickly as possible to keep them fresh and utilizing a gene-editing tool known as CRISPR to remove a gene. The cells must be sent back in liquid nitrogen to keep them frozen until they are needed. Patients must undergo chemotherapy before receiving their modified cells via IV, spending weeks in the hospital. The process can take several months.
Challenges
Great Challenges
These stark realities make the cost of gene therapies an insurmountable barrier, according to experts. The cost of gene therapies for sickle cell disease in the United States is $3.1 million and $2.2 million, although the cost of gene therapies can vary from country to country. The procedure for administering the treatments is also a significant obstacle. Patients must go to the hospital, where stem cells are extracted from their blood in a process that requires specialized equipment. One of the treatments requires sending the cells to a lab as quickly as possible to keep them fresh and using a gene-editing tool known as CRISPR to remove a gene. The cells must be sent back in liquid nitrogen to keep them frozen until they are used. Patients must undergo chemotherapy before receiving their modified cells through an IV, spending weeks in the hospital. The process can take several months.
Great Challenges
These stark realities make the cost of gene therapies an insurmountable barrier, according to experts. The cost of gene therapies for sickle cell disease in the United States is $3.1 million and $2.2 million, although the cost of gene therapies can vary from country to country. The procedure for administering the treatments is also a significant obstacle. Patients must go to the hospital, where stem cells are extracted from their blood in a process that requires specialized equipment. One of the treatments requires sending the cells to a lab as quickly as possible to keep them fresh and using a gene-editing tool known as CRISPR to remove a gene. The cells must be sent back in liquid nitrogen to keep them frozen until they are used. Patients must undergo chemotherapy before receiving their modified cells through an IV, spending weeks in the hospital. The process can take several months.
Great Challenges
These stark realities make the cost of gene therapies an insurmountable barrier, according to experts. The cost of gene therapies for sickle cell disease in the United States is $3.1 million and $2.2 million, although the cost of gene therapies can vary from country to country. The procedure for administering the treatments is also a significant obstacle. Patients must go to the hospital, where stem cells are extracted from their blood in a process that requires specialized equipment. One of the treatments requires sending the cells to a lab as quickly as possible to keep them fresh and using a gene-editing tool known as CRISPR to remove a gene. The cells must be sent back in liquid nitrogen to keep them frozen until they are used. Patients must undergo chemotherapy before receiving their modified cells through an IV, spending weeks in the hospital. The process can take several months.
Great Challenges
These stark realities make the cost of gene therapies an insurmountable barrier, according to experts. The cost of gene therapies for sickle cell disease in the United States is $3.1 million and $2.2 million, although the cost of gene therapies can vary from country to country. The procedure for administering the treatments is also a significant obstacle. Patients must go to the hospital, where stem cells are extracted from their blood in a process that requires specialized equipment. One of the treatments requires sending the cells to a lab as quickly as possible to keep them fresh and using a gene-editing tool known as CRISPR to remove a gene. The cells must be sent back in liquid nitrogen to keep them frozen until they are used. Patients must undergo chemotherapy before receiving their modified cells through an IV, spending weeks in the hospital. The process can take several months.
Challenges
High Costs
These stark realities make the costs of gene therapies a hurdle that cannot be overcome, according to experts. The cost of gene therapies for sickle cell disease in the United States is $3.1 million and $2.2 million, although the cost of gene therapies can vary from country to country. The process of administering the therapies is also a significant hurdle. Patients must go to the hospital, where stem cells are removed from their blood in a process that requires specialized equipment. One of the therapies requires sending the cells to a lab as quickly as possible to keep them fresh and using a gene-editing tool known as CRISPR to remove a gene. The cells must be sent back in liquid nitrogen to keep them frozen until they are used. Patients must undergo chemotherapy before receiving their modified cells through an intravenous line and spend weeks in the hospital. The process can take several months.
Major Challenges
These stark realities make the costs of gene therapies a hurdle that cannot be overcome, according to experts. The cost of gene therapies for sickle cell disease in the United States is $3.1 million and $2.2 million, although the cost of gene therapies can vary from country to country. The process of administering the therapies is also a significant hurdle. Patients must go to the hospital, where stem cells are removed from their blood in a process that requires specialized equipment. One of the therapies requires sending the cells to a lab as quickly as possible to keep them fresh and using a gene-editing tool known as CRISPR to remove a gene. The cells must be sent back in liquid nitrogen to keep them frozen until they are used. Patients must undergo chemotherapy before receiving their modified cells through an intravenous line and spend weeks in the hospital. The process can take several months.
Major Challenges
These stark realities make the costs of gene therapies a hurdle that cannot be overcome, according to experts. The cost of gene therapies for sickle cell disease in the United States is $3.1 million and $2.2 million, although the cost of gene therapies can vary from country to country. The process of administering the therapies is also a significant hurdle. Patients must go to the hospital, where stem cells are removed from their blood in a process that requires specialized equipment. One of the therapies requires sending the cells to a lab as quickly as possible to keep them fresh and using a gene-editing tool known as CRISPR to remove a gene. The cells must be sent back in liquid nitrogen to keep them frozen until they are used. Patients must undergo chemotherapy before receiving their modified cells through an intravenous line and spend weeks in the hospital. The process can take several months.
Major Challenges
These stark realities make the costs of gene therapies a hurdle that cannot be overcome, according to experts. The cost of gene therapies for sickle cell disease in the United States is $3.1 million and $2.2 million, although the cost of gene therapies can vary from country to country. The process of administering the therapies is also a significant hurdle. Patients must go to the hospital, where stem cells are removed from their blood in a process that requires specialized equipment. One of the therapies requires sending the cells to a lab as quickly as possible to keep them fresh and using a gene-editing tool known as CRISPR to remove a gene. The cells must be sent back in liquid nitrogen to keep them frozen until they are used. Patients must undergo chemotherapy before receiving their modified cells through an intravenous line and spend weeks in the hospital. The process can take several months.
Major Challenges
Big Challenges
These stark realities make the cost of gene therapies an insurmountable barrier, according to experts. The cost of gene therapies for sickle cell disease in the United States is $3.1 million and $2.2 million, although the cost of gene therapies can vary from country to country. The process of administering the therapies is also a significant hurdle. Patients must go to the hospital, where stem cells are removed from their blood in a process that requires specialized equipment. One of the treatments requires sending the cells to a lab as quickly as possible to keep them fresh and using a gene-editing tool known as CRISPR to remove a gene. The cells must be sent back in liquid nitrogen to keep them frozen until they are used. Patients must undergo chemotherapy before receiving their modified cells via IV, spending weeks in the hospital. The process can take several months.
Big Challenges
These stark realities make the cost of gene therapies an insurmountable barrier, according to experts. The cost of gene therapies for sickle cell disease in the United States is $3.1 million and $2.2 million, although the cost of gene therapies can vary from country to country. The process of administering the therapies is also a significant hurdle. Patients must go to the hospital, where stem cells are removed from their blood in a process that requires specialized equipment. One of the treatments requires sending the cells to a lab as quickly as possible to keep them fresh and using a gene-editing tool known as CRISPR to remove a gene. The cells must be sent back in liquid nitrogen to keep them frozen until they are used. Patients must undergo chemotherapy before receiving their modified cells via IV, spending weeks in the hospital. The process can take several months.
Big Challenges
These stark realities make the cost of gene therapies an insurmountable barrier, according to experts. The cost of gene therapies for sickle cell disease in the United States is $3.1 million and $2.2 million, although the cost of gene therapies can vary from country to country. The process of administering the therapies is also a significant hurdle. Patients must go to the hospital, where stem cells are removed from their blood in a process that requires specialized equipment. One of the treatments requires sending the cells to a lab as quickly as possible to keep them fresh and using a gene-editing tool known as CRISPR to remove a gene. The cells must be sent back in liquid nitrogen to keep them frozen until they are used. Patients must undergo chemotherapy before receiving their modified cells via IV, spending weeks in the hospital. The process can take several months.
Big Challenges
These stark realities make the cost of gene therapies an insurmountable barrier, according to experts. The cost of gene therapies for sickle cell disease in the United States is $3.1 million and $2.2 million, although the cost of gene therapies can vary from country to country. The process of administering the therapies is also a significant hurdle. Patients must go to the hospital, where stem cells are removed from their blood in a process that requires specialized equipment. One of the treatments requires sending the cells to a lab as quickly as possible to keep them fresh and using a gene-editing tool known as CRISPR to remove a gene. The cells must be sent back in liquid nitrogen to keep them frozen until they are used. Patients must undergo chemotherapy before receiving their modified cells via IV, spending weeks in the hospital. The process can take several months.
Big Challenges
Big
These stark realities make the cost of gene therapies an insurmountable barrier, according to experts. The cost of gene therapies for sickle cell disease in the United States is $3.1 million and $2.2 million although the cost of gene therapies can vary from country to country. The process of administering the therapies is also a major obstacle. Patients must go to the hospital, where stem cells are removed from their blood in a process that requires specialized equipment. One treatment requires sending the cells to a laboratory as quickly as possible to keep them fresh and using a gene-editing tool known as CRISPR to remove a gene. The cells must be sent back in liquid nitrogen
Source: https://apnews.com/article/sickle-cell-gene-therapy-crispr-global-0a76d10be53462d57e9d3cc07d15fc02
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