Sts

• Gene therapy is a method for treating or curing diseases by changing a person’s DNA.
• Other hematopoietic diseases include hemolytic anemia, aplastic anemia, and paroxysmal nocturnal hemoglobinuria.
• Gene therapy has been around for almost 50 years and is now the norm for some surgeries like bone marrow transplants.
• Gene therapy has advanced significantly over the past ten years and has been shown to be successful in treating conditions including stroke, autism, Parkinson’s, diabetes, spinal cord injury, etc.
• Repufranio Eriguel of La Union introduced House Bill No. 212 in 2013 which calls for the creation of a “bioethics advisory board” to create “ethical standards” for stem cell therapy.
• The goals of Gene Therapy are to turn the nation into a hub for open technology innovation in stem cell research and encourage investment and creation of jobs.
• Gene therapy is an experimental technique that uses genes to treat or prevent diseases, allowing doctors to insert a gene into a patient’s cells instead of using drugs or surgery.
• Several approaches to gene therapy include replacing a mutated gene that causes diseases with a healthy copy of the gene, inactivating or “knocking out” a mutated gene that is functioning properly, and introducing a new gene into the body to help fight a disease.
• Plasmid DNA are circular DNA molecules that can be genetically engineered to carry therapeutic genes into human cells.
• Viral vectors are derived from viruses and have a natural ability to deliver genetic material into cells, making them a type of gene therapy product.
• Bacterial vectors are modified bacteria that can be used as vectors (vehicles) to carry therapeutic genes into human tissues.
• Human gene editing technology is used to disrupt harmful genes or repair mutated genes.
• Bone marrow transplant and blood stem transplantation are used to treat leukemia and lymphoma.
• Blood collected through leukapheresis is a process where blood is drawn from the patient, passed through a machine that selects only the stem cells and returns all other components of the blood back to the patient.
• Mucolipidoses (ML) are a group of inherited metabolic disorders that affect the body’s ability to carry out the normal turnover of various materials within cells.
• Sickle cell anemia can cause blood cells to slow or block blood flow.
• Alpha Thalassemia Major is a program designed to address the complex diagnostic, prenatal, intrauterine, and perinatal management issues affecting a family with the disorder.
• Hemoglobinopathies are a group of disorders passed down through families (inherited) in which there is an abnormal production or structure of the hemoglobin molecule, with Sickle cell disease (SCD) being one such blood disorder.
• Bone marrow harvested by aspiration is used to treat patients undergoing chemotherapy.
• Beta Thalassemia Major is a blood disorder that reduces the production of hemoglobin, an iron-containing protein in red blood cells that carries oxygen to cells throughout the body.
• Sickle cell anemia affects the shape of red blood cells which are usually round and flexible, so they move easily through blood vessels, but in sickle cell anemia, some red blood cells are shaped like sickles or crescent moons.
• Mucolipidoses (ML) have an autosomal recessive pattern of inheritance.
• Mucopolysaccharidoses are a group of inherited metabolic diseases caused by the absence or malfunctioning of certain enzymes the body needs to break down molecules called glycosaminoglycans.
• In Utero Hematopoietic Stem Cell Transplantation (IUHCT) was developed as a strategy to treat genetic diseases that addresses the challenges associated with postnatal transplantation.
• Adipose stem cells are used for clinical trials for the treatment of diabetes mellitus, liver disease, corneal lesions, and articular and cutaneous lesions, among others.
• According to doctor-lawyer Samuel Bernal, an expert in the fields of regenerative medicine and regulatory law, stem cell therapy is not a cure to all medical procedures.
• Adipose tissues have the capacity of self-renewal and are harvested by liposuction.
• Patient-derived cellular gene therapy products involve removing, genetically modifying (often using a viral vector), and then returning the cells to the patient.
• Ex vivo gene therapy is a type of gene therapy where the cells are modified outside the body and delivered back to the patient.
• Vivo gene therapy is a type of gene therapy where the genetic modification of the cell takes place inside the body.
• Gene therapy that involves the immune system may cause an unwanted immune system reaction, affecting healthy cells that resemble the disease cells, potentially causing damage to healthy cells.
• There are three sources of autologous adult stem cells: bone marrow, blood, and umbilical cord blood.
• Gene therapy is being investigated for a number of rare, often fatal diseases using gene-modified blood stem cells with a goal of permanently correcting the underlying cause of disease.
• When a viral vector is used in gene therapy, there may be a risk that the virus could cause an infection, causing the person to have a weak immune system.
• Germline gene therapy prevents its side effects by altering, deleting, or adding specific genes to remedy an illness, or more frequently, get rid of undesired features.
• Therapeutics, a gene therapy company, outlines five ways gene therapy can cure, stop, or slow a disease: cancer, vision, movement, infectious diseases, and rare diseases.
• The immune reaction mediated by gene therapy can affect the wrong cell type instead of the intended target cells.
• Germline gene therapy is the process of transferring a portion of DNA to cells that make eggs or sperm.
• Stem cells have also been tapped to replace or control defective cells in patients who have certain diseases or defects.
• Stem cells are special type of cells in the body that have the ability to differentiate into other cell types, allowing them to replace cells that have died.