Scientists in Uganda, East Africa and Africa are now collaborating with their colleagues in USA and UK in research work using gene therapy for sickle cell patients and people living with HIV/Aids.

Scientists define Gene therapy as a technique that modifies a person's genes to treat or cure a specific disease.

Gene therapies can work by several mechanisms including replacing a disease causing gene with a healthy copy of the gene and inactivating a disease causing gene that is not functioning properly among others.

Background

Experts explain that gene therapy technology works well by using genes and deoxyribonucleic acid (DNA).

Genes include the Mendelian gene which is a basic unit of heredity and the molecular gene is a sequence of nucleotides in DNA that is transcribed to produce a functional RNA

DNA is the material that carries all the information about how living things will look and function. It supplies the genetic instructions that tell the body how to develop, live and reproduce.

Therefore since sickle cell and HIV/Aids are known to be diseases for developing countries especially in Africa and Uganda inclusive with no cure, scientists across the globe in collaborative effort are currently researching using gene therapy technology to find a cure for people suffering these diseases.

Scientists in USA are already in advanced stages of the research where they have products with products already being used.

They are already collaborating with scientists in Uganda where the initiative is led by scientists at the Joint Clinical Research Center (JCRC) who are currently preparing to implement the research activities in Uganda.

The facilities are already in place including laboratory equipment and research work will kick off from 2024.

This was revealed to a group of science journalists during a monthly biocafe in Uganda’s Kampala recently.

The Deputy Director of JCRC Dr Francis Ssalli took the journalists through the topic “Preparedness for gene therapy research and clinical services for the health and wellbeing of Ugandans” and below are the details.

Current Gene Therapy research Pattern

According to Dr Ssali sickle cell and HIV/Aids are a major challenge in Africa with Uganda ranking number 5 in sickle cell pandemic after Nigeria, Democratic Republic of Congo and Tanzania

Apparently the genetic engineering law which covered mainly issues related to agriculture Technology is now being reviewed by Uganda's Parliament to include aspects of advanced research in the health sector using gene therapy technology to allow scientists in the health sector to carry out research.

The aim is to get a cure for sickle cell and HIV/ Aids patients within the country and which is affordable. Currently patients who can afford are taken to countries such as India and USA to sick for sickle cell treatment using gene therapy technology.

Mr Ronald Jagwe from Uganda National Council for Science and Technology noted that Health care, environment  including agriculture are now key areas to be considered o=in modifying biosecurity laws

“Scientists are now prepared to apply modification technologies including issues of biosecurity and our scientists in the health sector are researching on use of gene therapy to address health challenges people are faced with,”

Dr Ssali notes that “what we know in health is that people are given medication either using injectable or tablets. Today we are advancing by using cells which can be obtained from another person to administer treatment. Colleagues from the agriculture sector have advanced in using these technologies where seed can be modified to increase yields,”

He adds that in the health sector scientists have not utilized human seed for modification to obtain cure but gene therapy has come on to address this incurable diseases.

Although trials are still ongoing and the long-term effects are still unclear, promising results show the potential for gene therapy as a treatment option.

Red blood cells contain haemoglobin which is a molecule that delivers oxygen to the body’s tissues which sickle cell patients’ lack.

In sickle cell disease, a problem with haemoglobin means that the red blood cells are C shaped like a sickle.

As a result, they can stick in the cardiovascular system. They are also unable to deliver oxygen effectively. This can affect the body in various ways.

Currently the only cure for sickle cell disease is a stem cell transplant, but organizations such as the Sickle Cell Disease Association of America are working to raise awareness and encourage funding for more research into this condition.

According to  a US based Genetic Home Reference, sickle cell disease affects people whose ancestors come from Africa, Mediterranean countries such as Greece, Turkey and Italy, the Arabian Peninsula, India, and Spanish-speaking regions in South America, Central America and parts of the Caribbean.

Types

HbSS is wher a person inherits two sickle cell genes, one from each parent. They will have sickle cell anemia, which is the most severe type of sickle cell disease.

HbSC is where a person inherits a sickle cell gene from one parent. From the other parent, they inherit a gene that results in another type of abnormal hemoglobin. This type is usually less severe than HbSS.

HbS beta-thalassemia is where a person inherits a sickle cell gene from one parent and a gene for beta-thalassemia, another type of anemia.

Sickle cell trait: is where if a person has only one sickle cell gene, they will not have sickle cell disease, but they can pass that gene to their children.

Statistical Data

According to the Centers for Disease Control and Prevention (CDC), about100, 000 people in the United States have sickle cell disease. Around 1 in 365 Black Americans are born with the disease, and 1 in 13 are born with the trait. Sickle cell disease affects about 1 in 16,300 Hispanic Americans.

The 2008 United Nations report estimates that there are between 20 and 25 million people worldwide living with SCD, of which 12–15 million live in Africa. The disease appears to be more likely in areas where is common  

Apparently Uganda is rated number with sickle cell prevalence in Africa after Nigeria, Democratic Republic of Congo, Tanzania and Sudan.

Symptoms

They include yellowing of the skin and white eyes, fatigue, pain and swelling in the hands and feet, acute chest syndrome, vision loss, enlarged spleen, liver, heart, or kidney damage, malnutrition in the young and infertility in males because causing prolonged painful  erection leading to high blood pressure and borne joint damage.

Medication

Apparently the drugs include Hydroxyurea (Hydrea) which helps ensure the supply of oxygen to the body.

Others are L-glutamine oral powder (Endari) which helps reduce the number of sickle cells, Voxelotor (Oxbryta) helps to boost haemoglobin supply and Crizanlizumab-tmca which helps reduce pain.

Gene therapy technology application  

According to Dr Ssali clinical trial have been in sickle in USA patients and there is treatment in place

This is done by isolating stem cells using bio vectors where virus that enter the cells will deliver stem cells. The gene the patient is obtained and cloned in the Laboratory. The virus will then infect the cells and the vector goes through the pours to deliver the required protein.

The cells that are isolated will get processed in 4 days and this is injected in the patient.

These process can be used to treat sickle cell which affects lymphocytes at the throat and this is done using T- cells where the t=gland will get trained to fight the disease.

Gene therapy application on HIV/Aids patients

Dr Ssali contends that the same process scientists go through modifying cells to treat sickle cell is done for HIV/ Patients.

“For HIV/AIDS we use are currently using anti-retroviral drugs as treatment for patients. Gene therapy technology use for started in US, India and UK and the first generation trials for cure is almost complete. This is a step already because in Uganda in 1980’s scientists were doing nothing but we have prepared the laboratory equipment and in 2030 we should be able to get a cure,”

So far scientists in USA have done 6 clinical trials using the CRISPR- Cas9 enzyme modification where cells were removed from a patient for evaluation.