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Adenoviruses and adeno-associated viruses (AAVs) are commonly used in gene therapy for Duchenne muscular dystrophy (DMD). These viruses are used as vectors to deliver the therapeutic gene to muscle cells to replace the mutated gene responsible for DMD. Additionally, lentiviruses have been investigated as potential vectors for gene therapy in DMD.
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How is DMD treated?
Duchenne muscular dystrophy (DMD) is usually treated with corticosteroids to help slow down muscle degeneration, physical therapy to maintain mobility and function, and medications to manage symptoms such as heart problems. Gene therapy and other new treatments are being developed to address the underlying genetic cause of DMD.
Which type of disorder is most difficult to correct by gene therapy?
Multifactorial disorders, such as heart disease and diabetes, are the most difficult to correct with gene therapy. These disorders involve a combination of genetic, environmental, and lifestyle factors, making it challenging to target a single gene for treatment. Additionally, the complexity of these disorders increases the risk of unintended consequences from gene therapy.
Is duchenne muscular dystrophy curable?
Theres no proper known cure for DMD, but its treatment is based on making the signs n symptoms better, however the choice of treatments are steroids Prednisol and Transplantation of Embryonic Stem Cells. I hope this answer helps! All the best! Dr. Malik! ** 7/2/09 ** Link added below showing new treatment for DMD.
What problems can occur in the musculoskeletal system?
Lots.Botulism or severe food poisoning, can lead to paralysisthere's muscular dystrophy, an inherited gene tetanus which results in muscle spasms and rigiditymyastrenia gravis(not so sure about the spelling) auto immune disease that causes muscular weaknessspasms,cramps,strains,pulls,tears,the list goes on.
What is Duchenne Muscular Dystrophy?
Duchenne muscular dystrophy (DMD) is a severe recessive X-linked form of muscular dystrophy characterized by rapid progression of muscle degeneration, eventually leading to loss of ambulation and death. This affliction affects one in 3500 males, making it the most prevalent of muscular dystrophies.A:A very simple explanation of Duchenne Muscular Dystrophy (DMD) Duchenne Muscular Dystrophy is an X chromosome-linked, inherited, progressive, muscle wasting disease, which affects approximately one out of 3,500 male children.For explaining the nature of Duchenne Muscular Dystrophy (DMD), we might start with the chromosomes.Chromosomes are specifically folded DNA molecules. We speak of chromosomes rather than DNA, because in this form the DNA molecules are visible under the microscope, therefore, distinguishable from each other.In normal circumstances we have 46 nuclear chromosomes. In certain conditions, such as cancer (for example in cancerous 'HeLa cells'), the number of chromosomes could be different. The 46 chromosomes include 2 sex chromosomes as well. We inherit 23 nuclear chromosomes (22 plus 1 sex chromosome) from each of our parent.Females have two X chromosomes (XX), males have one X and one Y (XY). They each contribute one to their child. Since the mother has two X-es, she passes down one X, therefore the father's chromosomes decides the child's sex.Duchenne muscular dystrophy arises from the mother's defective sex chromosome. The disease is a recessive trait, meaning only one of the mothers' two XX sex chromosomes is defective and the other X chromosome is able to compensate.For this reason, if she passes the disease-carrying chromosome to her daughter, the daughter's disease-free paternal X chromosome would compensate. The girl will carry, and potentially be able to pass, the faulty chromosome to her child/children, but, she, herself, will not be at all, or just mildly, affected.If, however, her son gets the disease-carrying chromosome, he will not have that opportunity; due to his Y chromosome from his father, he cannot compensate. Unfortunately, he will get the full-blown disease.While the majority of the disease are inherited from the mother, as detailed above, 'spontaneous mutations', when the damage to the gene happens in the male child, is also possible.So, what is the role of the X chromosome in that disease?The long DNA molecules, therefore their tightly packed forms, the chromosomes, have specific areas, called 'genes'. Every chromosome has its 'own' specific genes on their 'own' specific areas. Those genes have the 'blueprints', the instructions, for the body to build proteins. Proteins are compounds, specific substances, which are essential to life; they are vital for every function, feature, and aspects of the body.If a gene is faulty, inherited from either or both parents, (Duchenne muscular dystrophy is inherited only from the mother), or damaged/changed by environmental factors (like radiation, chemicals, or others), also by life style (smoking for example), then it cannot provide a correct instruction to assemble a properly working protein.The fault arise from mistakes in the instruction; some parts are missing, others are repeated, and some are changed. It is like someone is building a cupboard. If the instruction gives the wrong dimensions for some parts or even omit some others parts, then it would be impossible to build a proper cupboard.Neither can the body assemble the protein, called 'dystrophin', from the improper instruction given by the defective Dystrophin gene of the X-chromosome.Dystrophin is a muscle protein with mechanical and protective functions. It might involve in cell to cell communications, as well. Due to the lack of dystrophin protein the muscles became weaker, damage easily, and gradually break down, waste away.Symptoms (difficulty in sitting unaided, delayed start for walking, frequent stumbling and falling, difficulty in getting up, for example), could start to appear as early as one year of age, and the disease quickly progresses to the stage when the child needs a wheelchair (around 8-12 years of age).Since the disease weakens not only the skeletal muscles, but all muscles, heart rhythm and the pumping ability of the heart are also affected. Likewise, breathing becomes difficult and needs assistance as the disease progresses to those involved muscles.Lifespan, unfortunately, is not expected to extend beyond 30 years. Cure is not possible at the present.Treatments are aimed to delay the wastage, steroid medication (with Vitamin D and calcium supplement to counteract its possible bone weakening side-effect), physiotherapy, and water exercises, possibility of low frequency electrical muscle stimulation, among others.Improving the quality of the patients' life includes corrective bone surgery (bone deformities occurs frequently) to improve posture and comfort, braces, use of wheelchair (manual then electric), heart medications/treatments, assistance with breathing (ventilator, masks, others), - education/information/help to parents and carers, for example.Research is ongoing, internationally, on several possible aspects; a few examples are:· 'Gene bandage', specifically designed for the protein assembling process to 'skip over' the faulty segment of the gene, enabling the production of dystrophin,· Implantation of stem cells,· 'utrophin-based therapy', Utrophin is another muscle protein with similar function. Research going on to use it for compensate for the lack of dystrophin,· Researching a drug (recently trialled in Diabetes Type 2 research, as well), which, also, could increase the level of HSP72 (a specific protein). HSP72 expected to improve muscle function, decrease muscle break down, and increase lifespan by 20 %,· and an older research into low frequency electrical muscle stimulation.
What has the author Dongsheng Duan written?
Dongsheng Duan has written: 'Muscle gene therapy' -- subject(s): Duchenne Muscular Dystrophy, Laboratory Manuals, Muscles, Genetics, Diseases, Gene Therapy, Gene therapy, Therapy, Laboratory manuals, Methods
Who first studied muscular dystrophy?
The University of Pittsburgh reported the first study to achieve success with gene therapy for the treatment of congenital muscular dystrophy (CMD) in mice.
How can you get Muscular Dystrophy?
There is no known cure for muscular dystrophy, although Eastern philosophies believe that humans can heal many illnesses and conditions by "balancing" the body.In Muscular Dystrophy, prolonged inactivity (such as bed rest and even sitting for long periods) can worsen the disease. Physical therapy, occupational therapy, orthotic intervention (e.g., ankle-foot orthosis), speech therapy and orthopedic instruments (e.g., wheelchairs and standing frames) may be helpful.Occupational therapy assists the individual with MD in engaging in his/her activities of daily living (self-feeding, self-care activities, etc.) and leisure activities at the most independent level possible
Scientists are using gene therapy to manage what?
Cystic fibrosis and muscular dystrophy. There are perhaps more.
How is DMD treated?
Duchenne muscular dystrophy (DMD) is usually treated with corticosteroids to help slow down muscle degeneration, physical therapy to maintain mobility and function, and medications to manage symptoms such as heart problems. Gene therapy and other new treatments are being developed to address the underlying genetic cause of DMD.
Can you cure Muscular dystrophy?
There is no known cure for muscular dystrophy, although Eastern philosophies believe that humans can heal many illnesses and conditions by "balancing" the body.In Muscular Dystrophy, prolonged inactivity (such as bed rest and even sitting for long periods) can worsen the disease. Physical therapy, occupational therapy, orthotic intervention (e.g., ankle-foot orthosis), speech therapy and orthopedic instruments (e.g., wheelchairs and standing frames) may be helpful.Occupational therapy assists the individual with MD in engaging in his/her activities of daily living (self-feeding, self-care activities, etc.) and leisure activities at the most independent level possible
Which type of disorder is most difficult to correct by gene therapy?
Multifactorial disorders, such as heart disease and diabetes, are the most difficult to correct with gene therapy. These disorders involve a combination of genetic, environmental, and lifestyle factors, making it challenging to target a single gene for treatment. Additionally, the complexity of these disorders increases the risk of unintended consequences from gene therapy.
Is duchenne muscular dystrophy curable?
Theres no proper known cure for DMD, but its treatment is based on making the signs n symptoms better, however the choice of treatments are steroids Prednisol and Transplantation of Embryonic Stem Cells. I hope this answer helps! All the best! Dr. Malik! ** 7/2/09 ** Link added below showing new treatment for DMD.
What conditions does Trager psychophysical integration therapy treat?
Psychophysical integration therapy has been helpful in relieving muscle discomfort in patients afflicted with polio, muscular dystrophy, Parkinson's disease, multiple sclerosis, post-stroke trauma, and psychiatric disturbances
Muscular dystrophy?
DefinitionDuchenne muscular dystrophy is an inherited disorder that involves rapidly worsening muscle weakness.Alternative NamesPseudohypertrophic muscular dystrophy; Muscular dystrophy - Duchenne typeCauses, incidence, and risk factorsDuchenne muscular dystrophy is a rapidly-worsening form of muscular dystrophy. Other muscular dystrophies (including Becker's muscular dystrophy) get worse much more slowly.Duchenne muscular dystrophy is caused by a defective gene for dystrophin (a protein in the muscles). However, it often occurs in people without a known family history of the condition.Because of the way the disease is inherited, males are more likely to develop symptoms than are women. The sons of females who are carriers of the disease (women with a defective gene but no symptoms themselves) each have a 50% chance of having the disease. The daughters each have a 50% chance of being carriers.Duchenne muscular dystrophy occurs in approximately 1 out of every 3,600 male infants. Because this is an inherited disorder, risks include a family history of Duchenne muscular dystrophy.SymptomsSymptoms usually appear before age 6 and may appear as early as infancy. They may include:FatigueMental retardation (possible, but does not worsen over time)Muscle weakness Begins in the legs and pelvis, but also occurs less severely in the arms, neck, and other areas of the bodyDifficulty with motor skills (running, hopping, jumping)Frequent fallsRapidly worsening weaknessProgressive difficulty walkingAbility to walk may be lost by age 12By age 10, the person may need braces for walking. By age 12, most patients are confined to a wheelchair.Signs and testsA complete nervous system (neurological), heart, lung, and muscle exam may show:Abnormal heart muscle (cardiomyopathy)Congestive heart failure or irregular heart rhythm (arrhythmias) -- rareDeformities of the chest and back (scoliosis)Enlarged calf muscles, which are eventually replaced by fat and connective tissue (pseudohypertrophy)Loss of muscle mass (wasting)Muscle contractures in the heels, legsMuscle deformitiesRespiratory disorders, including pneumonia and aspiration of food or fluid into the lungs (in late stages of the disease)Tests may include:Electromyography (EMG)Genetic testsMuscle biopsySerum CPKTreatmentThere is no known cure for Duchenne muscular dystrophy. Treatment aims to control symptoms to maximize quality of life. Gene therapy may become available in the future.Activity is encouraged. Inactivity (such as bedrest) can worsen the muscle disease. Physical therapy may be helpful to maintain muscle strength and function. Orthopedic appliances (such as braces and wheelchairs) may improve mobility and the ability to care for yourself.Support GroupsYou can ease the stress of illness by joining a support group where members share common experiences and problems. See muscular dystrophy - support group. The Muscular Dystrophy Association is an excellent source of information on this disease.Expectations (prognosis)Duchenne muscular dystrophy leads to quickly worsening disability. Death usually occurs by age 25, typically from lung disorders.ComplicationsCardiomyopathyCongestive heart failure (rare)DeformitiesHeart arrhythmias (rare)Mental impairment (varies, usually minimal)Permanent, progressive disability Decreased mobilityDecreased ability to care for selfPneumonia or other respiratory infectionsRespiratory failureCalling your health care providerCall your health care provider if:Your child has symptoms of Duchenne muscular dystrophySymptoms worsen, or new symptoms develop, particularly fever with cough or breathing difficultiesPreventionGenetic counseling is advised if there is a family history of the disorder. Duchenne muscular dystrophy can be detected with about 95% accuracy by genetic studies performed during pregnancy.ReferencesKliegman RM, Behrman RE, Jenson HB, Stanton BF. Muscular dystrophies. In: Kliegman RM, Behrman RE, Jenson HB, Stanton BF. Nelson Textbook of Pediatrics. 18th ed. Philadelphia, Pa:Saunders Elsevier; 2007:chap 608.
How is muscular dystrophy treated?
Muscular dystrophy (MD) is primarily managed through a multidisciplinary approach, focusing on improving quality of life and maintaining mobility. Treatments may include physical therapy to enhance strength and flexibility, occupational therapy for daily living skills, and the use of assistive devices like braces or wheelchairs. Medications, such as corticosteroids, can help slow muscle degeneration, while newer gene therapies and experimental treatments are being explored. Regular monitoring by healthcare professionals is essential to address complications and optimize care.
What has gene therapy shown promise in controlling?
Gene therapy has shown promise in controlling genetic disorders such as cystic fibrosis, sickle cell anemia, and muscular dystrophy. It has also shown potential in treating certain types of cancer and inherited eye disorders.
