At Aster DM Healthcare, we are at the forefront of pioneering groundbreaking gene therapy treatments that are transforming the lives of patients affected by genetic conditions like Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD).
Imagine a world where diseases once deemed untreatable, such as Spinal Muscular Atrophy (SMA), no longer carry a sentence of lifelong struggle and debilitation. Aster DM Healthcare is making strides towards this vision, with its groundbreaking gene therapy program offering a tangible beacon of hope. By successfully treating over 160 patients with SMA, Aster DM is rewriting the possibilities in genetic medicine.

How Gene Therapy works
Gene therapy addresses the root cause of genetic diseases—faulty genes. By introducing healthy genes into a patient’s cells, we can correct these genetic defects and restore normal function.
Each cell in the body contains a set of instructions—called genes—that tell the cell how to build essential proteins. When these genes have errors, they produce malfunctioning or missing proteins, leading to diseases like SMA and DMD.
Through gene therapy, we introduce healthy copies of the gene into the affected cells.
These genes begin to produce the correct proteins, restoring normal cell function and improving the patient’s health. This revolutionary approach has the potential to cure previously incurable diseases, transforming the landscape of genetic disorder treatment.
A Beacon for Gene Therapy in the Gulf

Our expertise extends far beyond borders. Patients from many countries—including Russia, Turkey, and beyond—seek out our life-changing treatments. We are proud to be one of the few non-US, non-European centers offering gene therapy for DMD, a progressive muscle-wasting disorder.
Our success stories are a testament to the dedication and expertise of our multidisciplinary team, who work collaboratively to push the boundaries of gene therapy and deliver exceptional, personalized care to each patient.
Consult a Specialist
Gene Therapy for Spinal Muscular Atrophy (SMA)
This revolutionary treatment has the potential to transform lives, enabling improved motor function and reducing disease progression.
Gene Therapy for Duchenne Muscular Dystrophy (DMD)
We’re here for you with personalized care and constant support at every step of your journey.
Have Questions? Get Answers
What is Gene Therapy?
In the broadest sense, gene therapy is the use of genetic material in the treatment or prevention of disease. The transferred genetic material changes how a single protein or group of proteins is produced by the cell. Gene therapy can be used to reduce levels of a disease-causing version of a protein, increase production of disease-fighting proteins, or to produce new/modified proteins.
What is cell therapy?
Cell therapy is the transfer of intact, live cells into a patient to help lessen or cure a disease. The cells may originate from the patient (autologous cells) or a donor (allogeneic cells). The cells used in cell therapy can be classified by their potential to transform into different cell types. Pluripotent cells can transform into any cell type in the body and multipotent cells can transform into other cell types, but their repertoire is more limited than that of pluripotent cells. Differentiated or primary cells are of a fixed type. The type of cells administered depends on the treatment.
What is CAR T cell therapy?
CAR T cell stands for chimeric antigen receptor (CAR) T cell therapy. This a way of modifying the patient’s own immune cells (T-cells) to express a receptor on their surface that recognizes structures (antigens) on the surface of malignant cells. Once the receptor binds to a tumor antigen, the T-cell is stimulated to attack the malignant cells.
What is the difference between gene therapy and cell therapy?
Gene therapy involves the transfer of genetic material, usually in a carrier or vector, and the uptake of the gene into the appropriate cells of the body. Cell therapy involves the transfer of cells with the relevant function into the patient.
Some protocols utilize both gene therapy and cell therapy. In this case, stem cells are isolated from the patient, genetically modified in tissue culture to express a new gene, expanded to sufficient numbers, and then returned to the patient.
Are there different types of gene therapy?
Almost any gene in the human genome can be targeted, so the potential for new therapies is immense. The five main therapeutic strategies are presented below. Currently, these techniques are mainly used to target specific populations of somatic cells.
Gene addition involves inserting a new copy of a gene into the target cells to produce more of a protein. Most often, a modified virus such as adeno-associated virus (AAV) is used to carry the gene into the cells. Therapies based on gene addition are being developed to treat many diseases, including adenosine deaminase severe combined immunodeficiency (ADA- SCID), congenital blindness, hemophilia, Leber’s congenital amaurosis, lysosomal storage diseases, X-linked chronic granulomatous disease, and many others.
Gene correction can be achieved by modifying part of a gene using recently-developed gene editing technology (e.g. CRISPR/cas9, TALEN or ZFN) to remove repeated or faulty elements of a gene, or to replace a damaged or dysfunctional region of DNA. The goal of gene correction is to produce a protein that functions in a normal manner instead of in a way that contributes to disease. It may be possible to use gene correction in the treatment of a wide range of diseases; recent experimental work has used gene editing technologies to extract HIV from the genome of affected laboratory mice and to excise the expanded region responsible for Huntington’s disease from the human gene.
Gene silencing prevents the production of a specific protein by targeting messenger RNA (mRNA; an intermediate required for protein expression from a gene) for degradation so that no protein is produced. mRNA exists in a single-stranded form in human and animal cells, whereas viruses have double-stranded RNA. Human and animal cells recognize double-stranded RNA as being viral in origin and destroy it to prevent its spread. Gene silencing uses small sequences of RNA to bind unique sequences in the target mRNA and make it double-stranded. This triggers the destruction of the mRNA using the cellular machinery that destroys viral RNA. Gene silencing is an appropriate gene therapy for the treatment of diseases where too much of a protein is produced. For example, too much tumor necrosis factor (TNF) alpha is often observed in the afflicted joints of rheumatoid arthritis patients. As TNF alpha is needed in small amounts in the rest of the body, gene silencing is used to reduce TNF alpha levels only in the affected tissue.
Reprogramming involves adding one or more genes to cells of a specific type to change the characteristics of those cells. This technique is particularly powerful in tissues where multiple cell types exist and the disease is caused by dysfunction in one type of cells. For example, type I diabetes occurs because many of the insulin-producing islet cells of the pancreas are damaged. At the same time, the cells of the pancreas that produce digestive enzymes are not damaged. Reprogramming these cells so that they can produce insulin would help heal type I diabetic patients.
Cell elimination strategies are typically used to destroy malignant (cancerous) tumor cells, but can also be used to target overgrowth of benign (non-cancerous) tumor cells. Tumor cells can be eliminated via the introduction of “suicide genes,” which enter the tumor cells and release a prodrug that induces cell death in those cells. Viruses can be engineered to have an affinity for tumor cells. These oncotropic viruses can carry therapeutic genes to increase toxicity to tumor cells, stimulate the immune system to attack the tumor, or inhibit the growth of blood vessels that supply the tumor with nutrients.
Are there different types of cell therapy?
The most common type of cell therapy is blood transfusion, and the transfusion of red blood cells, white blood cells, and platelets from a donor. Another common cell therapy is the transplantation of hematopoietic stem cells to create bone marrow which has been performed for over 40 years. As with gene therapy, cell therapy subtypes can be classified in different ways. This is currently no formal classification system for cell therapies. Here the different types of cells used for cell therapy have been classified by cell potency. Four types of pluripotent stem cells and four types of multipotent stem cells obtained from adult tissue are described.
Embryonic stem cells (ESCs). These are pluripotent stem cells derived from embryos. Generally, the embryos used to isolate stem cells are unused embryos generated from in vitro fertilization (IVF) for assisted reproduction. As ESCs are pluripotent they retain the ability to self-renew and to form any cell in the body. ESCs have the advantage of versatility due to their pluripotency, but the use of embryos in the development of therapeutic strategies raises some ethical concerns. In addition, stem cell lines generated from embryos are not genetically matched to the patient which can increase the chance that the transplanted cell is rejected by the patient’s immune system.
Induced pluripotent stem cells (iPSCs). A differentiated adult (somatic) cell, such as a skin cell is reprogrammed to return to a pluripotent state. These cells offer the advantage of pluripotency but without the ethical concerns of embryonic stem cells. iPSCs may also be derived from the patient and thus avoid the problem of immune rejection. iPSCs are produced by transforming the adult cell with a cocktail of genes usually delivered via a viral vector. While the efficiency of the process has been greatly improved since inception, the relatively low rate of reprogramming remains a concern. Another concern is that iPSCs are derived from adult cells and are therefore “older” than embryonic stem cells as evidenced by a higher rate of programmed cell death, lower rates of DNA damage repair and increased incidence of point mutations.
Nuclear transfer embryonic stem cells (ntESCs). These pluripotent cells are produced by transferring the nucleus from an adult cell obtained from the patient to an oocyte (egg cell) obtained from a donor. The process of transferring the nucleus reprograms the egg cell to pluripotency. As with iPSCs, the derived cells match the nuclear genome of the patient and are unlikely to be rejected by the body. However, the major advantage of this technique is that the resulting ntESCs carry the nuclear DNA of the patient alongside mitochondria from the donor, making this technique particularly appropriate for diseases where the mitochondria are damaged or dysfunctional. A drawback of ntESCs is that the process of generation is cumbersome and requires a donor oocyte. At the time of writing stem cell production using this technique has only been shown in lower mammals.
Parthenogenetic embryonic stem cells (pES). The final option for obtaining pluripotent cells is from unfertilized oocytes. Here the oocyte is treated with chemicals that induce embryo generation without the addition of sperm (parthenogenesis) and ESCs are harvested from the developing embryo. This technique generates ESCs that are genetically identical to the female patient. However, this method is in the early stages of development and it is not known if cells and tissues derived from parthenogenesis develop normally.
Hematopoietic stem cells (HSCs) are multipotent blood stem cells that give rise to all types of blood cells. HSCs can be found in adult bone marrow, peripheral blood, and umbilical cord blood.
Mesenchymal stem cells (MSCs) are multipotent cells present in multiple tissues including umbilical cord, bone marrow, and fat tissue. MSCs give rise to bone, cartilage, muscle, and adipocytes (fat cells) which promotes marrow adipose tissue.
Neural stem cells (NSCs). Adult neural stem cells are present in small number in defined regions of the mammalian brain. These multipotent cells replenish neurons and supporting cells of the brain. However, adult neural stem cells cannot be obtained from patients due to their location in the brain. Therefore, neural stem cells used for cell therapies are obtained from iPSCs or ESCs.
Epithelial stem cells. Epithelial cells are those that form the surfaces and linings of the body including the epidermis and the lining of the gastro-intestinal tract. Multipotent epithelial stem cells are found in these areas along with unipolar stem cells that only differentiate into one type of cell. Epithelial stem cells have been successfully used to regenerate the corneal epithelium of the eye.
Immune cell therapy. Cells that rapidly reproduce in the body such as immune cells, blood cells or skin cells can usually do so ex vivo given the right conditions. This allows differentiated, adult immune cells to be used for cell therapy. The cells can be removed from the body, isolated from a mixed cell population, modified and then expanded before return to the body. A recently developed cell therapy involves the transfer of adult self-renewing T lymphocytes which are genetically modified to increase their immune potency to kill disease-causing cells.
What risks are associated with gene therapy and cell therapy?
Risks of any medical treatment depend on the exact composition of the therapeutic agent and its route of administration. Different types of administration, whether intravenous, intradermal or surgical, have inherent risks.
Risks include the outcome that gene therapy or cell therapy will not be as effective as expected, possibly prolonging or worsening symptoms, or complicating the condition with adverse effects of the therapy. The expression of the genetic material or the survival of the stem cells may be inadequate and/or may be too short-lived to fully heal or improve the disease. Their administration may induce a strong immune response to the protein in the case of replacing proteins from genetic diseases. This immune response may become uncontrolled and lead to normal proteins or cells being attacked, as in autoimmune diseases. On the other hand, in the case of cancer or viral/fungal/bacterial infections, there may be an insufficient immune response, or the targeted cell or microorganism may develop resistance to the therapy. With the current generation of vectors in clinical trials, there is no way to “turn off” gene expression, if it seems to be producing unwanted effects.
In the case of retroviral or lentiviral vectors, integration of the genetic material into the patients’ DNA may occur next to a gene involved in cell growth regulation and the insertion may induce a tumor over time by the process called insertional mutagenesis.
High doses of some viruses can be toxic to some individuals or specific tissues, especially if the individuals are immune compromised.
Gene therapy evaluation is generally carried out after birth. There is little data on what effects this therapeutic approach might have on embryos, and so pregnant women are usually excluded from clinical trials.
Risks of cell therapy also include the loss of tight control over cell division in the stem cells. Theoretically, the transplanted stem cells may gain a growth advantage and progress to a type of cancer or teratomas. Since each therapy has potential risks, patients are strongly encouraged to ask questions of their investigators and clinicians until they fully understand the risks
How are genes delivered?
Scientists and clinicians use the following four methods to carry genetic material into the targeted cells.
Non-vector methods such as electroporation, passive delivery, and ballistic delivery. Simple strands of naked DNA or RNA can be pushed into cells using high voltage electroporation. This is a common technique in the lab. Naked DNA or RNA may also be taken up by target cells using a normal cellular process called endocytosis after addition to the medium surrounding the cells. Finally, sheer mechanical force can be utilized to introduce genetic material with an instrument called a “gene gun.”
Membrane-bound vesicles. Genetic material can be packaged into artificially-created liposomes (sacs of fluid surrounded by a fatty membrane) that are more easily taken up into cells than naked DNA/RNA. Different types of liposomes are being developed to preferentially bind to specific tissues. Recent work has utilized a subtype of membrane vesicles that are endogenously produced and released by cells (extracellular vesicles or “exosomes”) to carry small sequences of RNA into specific tissues.
Viral vectors. Viruses have an innate ability to invade cells. The symptoms of a cold are triggered by a cold virus entering the cells of the upper respiratory tract and hijacking the cell’s machinery to manufacture more virus. Viral vectors for gene therapy are modified to utilize the ability of viruses to enter cells after disabling the capability of the virus to divide. Different types of viruses have been engineered to function as gene therapy vectors. In the case of adeno-associated virus (AAV) and retrovirus/lentivirus vectors, the gene(s) of interest and control signals replace all or most of the essential viral genes in the vector so the viral vector does not replicate. For oncolytic viruses, such as adenovirus and herpes simplex virus, fewer viral genes are replaced and the virus is still able to replicate in a restricted number of cell types. Different types of viral vector preferentially enter a subset of different tissues, express genes at different levels, and interact with the immune system differently.
Gene therapy can be combined with cell therapy protocols. Cells are collected from the patient or matched donor and then purified and expanded in vitro. Scientists and clinicians then deliver the gene to the cells using one of the three methods described above. Those cells that express the therapeutic gene are then re-administered to the patient.
How does gene therapy work?
Put simply, gene therapy works by changing the genetic information of a population of cells in a way that alleviates or combats the cause or symptoms of a disease.
What are stem cells and where do they come from?
Stem cells are cells that can self-renew and can mature into at least one type of specialized cell. Stem cells can be isolated from many types of tissues. Embryonic stem cells are isolated from the inner mass of the blastocyst, an early stage of the embryo. Umbilical cord stem cells, often called cord blood stem cells, are isolated from the umbilical cord at the time of a baby’s birth.
Adult stem cells can be isolated from any type of adult tissue. The ease of isolation of adult stem cells depends on the accessibility of the tissue, the prevalence of stem cells in the tissue, the age of the patient, the presence of markers that aid stem cell isolation, and developed protocols for isolation and culture. It is also possible to convert a mature adult cell into a stem cell by introducing a mixture of transcription factors; these cells are referred to as induced pluripotent stem (iPS) cells.
What kinds of diseases do gene and cell therapy treat?
Characteristics of diseases amenable to gene therapy and cell therapy include those for which there is no effective treatment, those with a known cause (such as a defective gene), those that have failed to improve or have become resistant to conventional therapy, and/or cases where current therapy involves long term administration of an expensive therapeutic agent or an invasive procedure.
Gene therapy and cell therapy have the potential for high therapeutic gain for a broad range of diseases. An example would be those caused by a mutation in a single gene where an accessible tissue is available, such as bone marrow, and with the genetically modified cell ideally having a survival advantage. However, patients with similar symptoms may have mutations in different genes involved in the same biological process. For example, patients with hemophilia A have a mutation in blood clotting Factor VIII whereas patients with hemophilia B have a mutation in Factor IX. It is important to know which gene is mutated in a particular patient, as well as whether they produce an inactive protein which can help to avoid immune rejection of the normal protein.
Gene therapy and cell therapy also offer a promising alternative or adjunct treatment for symptoms of many acquired diseases, such as cancer, rheumatoid arthritis, diabetes, Parkinson’s disease, Alzheimer’s disease, etc. Cancer is the most common disease in gene therapy clinical trials. Cancer gene therapy focuses on eliminating the cancer cells, blocking tumor vascularization and boosting the immune response to tumor antigens. Many gene and cell therapy approaches are being explored for the treatment of a variety of acquired diseases.