• Cancers
    • Colorectal Cancer
    • Lymphoma
    • Penile Cancer
    • Uterine Cancer
    • Breast Cancer
    • Stomach Cancer
    • Bone Cancer
    • Cervical Cancer
    • Brain Cancer
    • Leukemia (Blood Cells Cancer)
    • Endometrial Cancer
    • Neuroblastoma
    • Kidney Cancer
  • Brain
    • Stroke
    • Dyslexia
    • Down Syndrome
    • Cerebral Palsy
    • Attention Deficit Hyperactivity Disorder (ADHD)
    • Alcoholism
    • Autism
    • Multiple Sclerosis
    • Epilepsy
    • Schizophrenia
    • Insomnia
    • Alzheimer’s Disease
    • Parkinson’s Disease
    • Migraine
    • Phobia
  • Heart
    • Hypertension
    • Endocarditis
    • Coronary Heart Disease
    • Myocardial Infarction
    • Arrhythmia
    • Angina
    • Enlarged heart
    • Pericarditis
    • Venous Thromboembolism (VTE)
    • Peripheral Arterial Disease
    • Ventricular Septal Defect
    • Hypotension
  • Urinary Health
    • Chronic Kidney Disease
    • Diabetic Nephropathy
    • Urinary Tract Infection
    • Prostatitis
    • Urinary Incontinence
    • Enlarged Prostate
    • Blood in Urine
    • Kidney Stones
    • Erectile Dysfunction
    • Cystitis
  • Eye
    • Myopia
    • Glaucoma
    • Dry Eyes
    • Conjunctivitis
    • Cataracts
    • Color Blindness
    • Macular Degeneration
    • Diabetic Retinopathy
    • Retinal Detachment
    • Keratoconus
  • Skin
    • Shingles
    • Rosacea
    • Eczema
    • Vitiligo
    • Psoriasis
    • Scabies
    • Acne
    • Dandruff
  • Women Health
    • Uterine Fibroids
    • Pregnancy
    • Polycystic Ovary Syndrome
    • Vaginitis
    • Endometriosis
    • Preeclampsia
    • Menstruation
    • Ovarian Cyst
    • Pelvic Inflammatory Disease
  • Digestive Health
    • Celiac Disease
    • Hemorrhoids
    • Achalasia
    • Diarrhea
    • Gastroesophageal Reflux Disease
    • Diverticulitis
    • Liver Cirrhosis
    • Irritable Bowel Syndrome
    • Constipation
    • Appendicitis
    • Ulcerative Colitis
    • Gallstones
    • Crohn’s Disease
  • Bone Health
    • Osteoarthritis
    • Osteoporosis
    • Cyst
    • Osteomyelitis
    • Sciatica
    • Fibromyalgia
    • Calcium Deficiency
    • Gout
    • Rickets in Children
    • Spinal Stenosis
  • Dental
    • Periodontal Disease
    • Dental Caries
    • Bad Breath
    • Sensitive Teeth
    • Dry Mouth
    • Supernumerary Teeth
    • Bruxism
    • Tooth Loss
    • Impacted Tooth
  • More
    • Hyperthyroidism
    • Rhinitis
    • Chronic Obstructive Pulmonary Disease (COPD)
    • Thalassemia
    • Hepatitis
    • Sinusitis
    • Acromegaly
    • Health and Wellness
    • Achondroplasia
    • Diabetes Mellitus
    • Asthma
    • Coronavirus Disease (Covid-19)
    • Sleep Apnea
    • Tuberculosis

Write For Us

What's Hot

How Big Are Kidney Stones?

February 3, 2021
Dyslexia

What Do Words Look Like to a Dyslexic Person?

February 3, 2021
Gallstones without a gallbladder image

Can you Get Gallstones without a Gallbladder?

July 12, 2022
Facebook Twitter YouTube
DiseaseFix – World's Largest Disease Information Center
  • Write For Us
Facebook Twitter YouTube
  • Cancers
    • Colorectal Cancer
    • Lymphoma
    • Penile Cancer
    • Uterine Cancer
    • Breast Cancer
    • Stomach Cancer
    • Bone Cancer
    • Cervical Cancer
    • Brain Cancer
    • Leukemia (Blood Cells Cancer)
    • Endometrial Cancer
    • Neuroblastoma
    • Kidney Cancer
  • Brain
    • Stroke
    • Dyslexia
    • Down Syndrome
    • Cerebral Palsy
    • Attention Deficit Hyperactivity Disorder (ADHD)
    • Alcoholism
    • Autism
    • Multiple Sclerosis
    • Epilepsy
    • Schizophrenia
    • Insomnia
    • Alzheimer’s Disease
    • Parkinson’s Disease
    • Migraine
    • Phobia
  • Heart
    • Hypertension
    • Endocarditis
    • Coronary Heart Disease
    • Myocardial Infarction
    • Arrhythmia
    • Angina
    • Enlarged heart
    • Pericarditis
    • Venous Thromboembolism (VTE)
    • Peripheral Arterial Disease
    • Ventricular Septal Defect
    • Hypotension
  • Urinary Health
    • Chronic Kidney Disease
    • Diabetic Nephropathy
    • Urinary Tract Infection
    • Prostatitis
    • Urinary Incontinence
    • Enlarged Prostate
    • Blood in Urine
    • Kidney Stones
    • Erectile Dysfunction
    • Cystitis
  • Eye
    • Myopia
    • Glaucoma
    • Dry Eyes
    • Conjunctivitis
    • Cataracts
    • Color Blindness
    • Macular Degeneration
    • Diabetic Retinopathy
    • Retinal Detachment
    • Keratoconus
  • Skin
    • Shingles
    • Rosacea
    • Eczema
    • Vitiligo
    • Psoriasis
    • Scabies
    • Acne
    • Dandruff
  • Women Health
    • Uterine Fibroids
    • Pregnancy
    • Polycystic Ovary Syndrome
    • Vaginitis
    • Endometriosis
    • Preeclampsia
    • Menstruation
    • Ovarian Cyst
    • Pelvic Inflammatory Disease
  • Digestive Health
    • Celiac Disease
    • Hemorrhoids
    • Achalasia
    • Diarrhea
    • Gastroesophageal Reflux Disease
    • Diverticulitis
    • Liver Cirrhosis
    • Irritable Bowel Syndrome
    • Constipation
    • Appendicitis
    • Ulcerative Colitis
    • Gallstones
    • Crohn’s Disease
  • Bone Health
    • Osteoarthritis
    • Osteoporosis
    • Cyst
    • Osteomyelitis
    • Sciatica
    • Fibromyalgia
    • Calcium Deficiency
    • Gout
    • Rickets in Children
    • Spinal Stenosis
  • Dental
    • Periodontal Disease
    • Dental Caries
    • Bad Breath
    • Sensitive Teeth
    • Dry Mouth
    • Supernumerary Teeth
    • Bruxism
    • Tooth Loss
    • Impacted Tooth
  • More
    • Hyperthyroidism
    • Rhinitis
    • Chronic Obstructive Pulmonary Disease (COPD)
    • Thalassemia
    • Hepatitis
    • Sinusitis
    • Acromegaly
    • Health and Wellness
    • Achondroplasia
    • Diabetes Mellitus
    • Asthma
    • Coronavirus Disease (Covid-19)
    • Sleep Apnea
    • Tuberculosis
DiseaseFix – World's Largest Disease Information Center
Home » Rare Diseases » Duchenne Muscular Dystrophy » Gene Therapy For DMD: Research, Benefits & Challenges

Gene Therapy For DMD: Research, Benefits & Challenges

Neha MittalBy Neha MittalUpdated:April 22, 20213 Mins Read
Facebook Twitter Pinterest LinkedIn Tumblr Email
Share
Facebook Twitter LinkedIn Pinterest Email
Contents Hide
1 Duchenne Muscular Dystrophy Gene Therapy
2 What is the objective of gene therapy for Duchenne muscular dystrophy (DMD)?
3 Research on gene therapy for Duchenne muscular dystrophy
4 Challenges with gene therapy as a treatment for DMD
5 What benefits can be achieved with gene therapy in future for DMD?

Duchenne Muscular Dystrophy Gene Therapy

Duchenne muscular dystrophy (DMD) is an X-linked muscle-weakening disease that is caused by mutations in the dystrophin gene. Gene therapy using highly functional microdystrophin genes and recombinant adeno-associated virus (rAAV) vectors is an attractive treatment strategy for DMD that is being researched and developed currently.

What is the objective of gene therapy for Duchenne muscular dystrophy (DMD)?

The primary objective of gene therapy is to cure a patient with DMD completely.

There is some research going on that is aimed for delivering a functional, full-size dystrophin gene to muscle cells using a harmless virus. Until now, research has been limited to delivering smaller mini- or micro-dystrophin genes due to restrictions in the size of the DNA fragment that the virus can accommodate.

See also: Duchenne Muscular Dystrophy (DMD): Causes, Signs, Symptoms, Diagnosis, and Treatment

See also: Duchenne Muscular Dystrophy Life Expectancy

Research on gene therapy for Duchenne muscular dystrophy

Professor George Dickson at Royal Holloway, University of London, is running a project funded by the Duchenne forum to deliver newer solutions for gene therapy to treat Duchenne muscular dystrophy.

In this project, he and his team will use multiple viruses each carrying a different part of the dystrophin gene. The different parts of the gene will assemble in the muscle cell to develop the blueprint to produce a full size dystrophin protein. This method, if successful, is expected to significantly improve the treatment strategy for Duchenne muscular dystrophy patients. It will also be effective in treating people with Becker muscular dystrophy.

A phase I clinical trial has also been done to test the safety of this approach in a small number of boys.

Another simple method for delivery can be the use of intravascular routes as capillaries surround all muscle fibers. However, a challenge with this approach is that vectors do not easily pass through vessel walls.

Recent progress in the area suggests that safe vectors will soon be available for dystrophin gene delivery to the muscles of patients. But, till date, there is no effective a method to deliver these vectors to all the muscles of the body. However, crude ways of delivery may be used that may target selected limb muscles to improve mobility and quality of life for patients. But, these methods are not generally applied to complex organs such as heart or diaphragm.

Challenges with gene therapy as a treatment for DMD

There are some challenges with gene therapy for duchenne muscular dystrophy, such as:

  • Gene therapy for Duchenne muscular dystrophy requires efficient delivery of a dystrophin expression vector to the muscles of the body.
  • The dystrophin gene is too big for the virus to carry, and it is not easy to carry the gene through the virus. The dystrophin gene is 2.4 Mb in size. Approaches are being developed to carry parts of the virus through separate genes.
  • Using a mini or micro sized dystrophin gene has its own limitations.
  • Muscle transduction must not cause toxic or immunological reactions that are harmful to a person and can lead to further muscle damage.
  • Innate and acquired immune defense mechanisms can block the extent of gene transfer, and in severe cases cause reactions that can result in death of a person too.

What benefits can be achieved with gene therapy in future for DMD?

The key benefit that gene therapy can provide is to offer the possibility of delivering a functional copy of the dystrophin gene to muscle cells where it could begin production of the dystrophin protein.

Neha Mittal
  • LinkedIn

Neha Mittal completed graduation in Biotechnology from Graphics Era University, Dehradun and Masters in Biotechnology from Amity University, Noida. In the past, she worked with Sharon Biomedicine Ltd., DUKES Products Ltd., and Forest Research Institute, Dehradun as researcher in biotechnology. Neha loves singing and dancing in leisure time. She also enjoys traveling and maintains a collection of great novels.

Follow-Up Reads

Duchenne Muscular Dystrophy Life Expectancy

April 22, 2021

Muscular Dystrophy Life Expectancy: Prognosis and Outlook

April 21, 2021

Duchenne Muscular Dystrophy (DMD): Causes, Signs, Symptoms, Diagnosis, Treatment, Survival

September 27, 2019

Leave A Reply Cancel Reply

Best Practices for staying Healthy: Strategies & Tactics
doctor with mask Best Practices for staying Healthy

Treatment for COPD in the Elderly Explained

By Kapil Sharma5 Mins Read

Chronic obstructive pulmonary disease (COPD) is a common and debilitating condition that affects the lungs,…

Lemon Juice Glass

Top 10 Home Remedies to Pass a Kidney Stone in 24 Hours

February 20, 2023
Runny Nose

Spike in H3N2 Influenza Cases in Delhi-NCR

February 16, 2023
Newborn child

Healing From a Stillbirth or Infant Loss: A Guide for Parents

February 14, 2023
Latest Posts
Brillia Helped Child During Class

How Brillia Has Helped My Child Focus During Class

June 6, 2023
Blue ceramic coffee cup

Home Detox and Cleansing Trends

June 2, 2023
CT Scan

Mastering the Art of Reading CT Scans for Cancer Diagnosis: Key Tips and Techniques

June 2, 2023
Depressant Effects

Is Alcohol a Stimulant: Separating Fact From Fiction

June 2, 2023
person showing magnifying eyeglasses

10 Essential Medical Equipment for Every Healthcare Facility

June 1, 2023
Subscribe To Our Newsletter
About Us
About Us

DiseaseFix is primarily known as an online publisher of disease-centric medical information related to human health and well-being. The information is published as disease modules accessible to users from across the world free of cost.

Email Us: contact@diseasefix.com

Our Picks
Breast Cancer

Moving Forward After Breast Cancer Surgery

December 30, 2022
Five Things Patients Experience Immediately After Breast Cancer Diagnosis

Five Things Patients Experience Immediately After Breast Cancer Diagnosis

April 23, 2021

Real Life Experiences About Coping With Cancer

April 22, 2021
Company

Disease Index

About Us

Our Team

Privacy Policy

Disclaimer

Contact Us

Write For Us

 

Facebook Twitter YouTube
© 2023 Diseasefix. All rights reserved.

Type above and press Enter to search. Press Esc to cancel.