Duchenne Muscular Dystrophy News and Research

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Duchenne muscular dystrophy (DMD) is a progressive muscle disorder that causes the loss of both muscle function and independence. DMD is perhaps the most prevalent of the muscular dystrophies and is the most common lethal genetic disorder diagnosed during childhood today. Each year, approximately 20,000 children worldwide are born with DMD (one of every 3,500 male children).

New Gene Therapy Approach Improves Muscle Strength in DMD Mice

Indiana University School of Medicine researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments for people with Duchenne muscular dystrophy (DMD).

29 Jul 2024

Harnessing Enzymes for Making RNA Therapeutics

While the COVID-19 vaccines introduced many people to RNA-based medicines, RNA oligonucleotides have already been on the market for years to treat diseases like Duchenne Muscular Dystrophy and amyloidosis.

15 Jul 2024

Review: Role of Alternative Splicing Defects in Major Neurodegenerative Diseases

Alternative splicing, a clever way a cell generates many different variations of messenger RNAs -; single-stranded RNAs involved in protein synthesis -; and proteins from the same stretch of DNA, plays an important role in molecular biology.

21 Jun 2023

Scarring on the Extracellular Matrix Causes Muscles to Stop Working in Duchenne Muscular Dystrophy

Muscles that ache after a hard workout usually don't hurt for long, thanks to stem cells that rush to the injured site along "collagen highways" within the muscle and repair the damaged tissue.

12 May 2023

The relationship between cancer and DMD gene expression discovered

Mutations of the gene encoding dystrophins have long been known to cause the debilitating muscle-wasting disease DMD, which affects one in every 5,000 boys born. People with the condition will usually only live into their 20s or 30s.

28 Feb 2023

Scientists receive $2.5 million NIH grant to define the role of lipid droplets in muscle stem cell function

A team of Purdue University scientists led by Shihuan Kuang has received a $2.5 million grant from the National Institutes of Health to define the role of lipid droplets in muscle stem cell function, a study with implications in both humans and livestock.

24 Feb 2023

Cell-derived therapy may help treat ventricular arrhythmia

Vesicles secreted from human heart cells may repair damaged tissue and prevent lethal heart rhythm disorders, according to a new study from investigators in the Smidt Heart Institute at Cedars-Sinai.

13 Apr 2022

Cellular therapy leads to meaningful functional improvements in DMD patients

A clinical trial at UC Davis Health and six other sites showed that a cellular therapy offers promise for patients with late-stage Duchenne muscular dystrophy (DMD), a rare genetic disorder causing muscle loss and physical impairments in young people.

From The University of California, Davis 14 Mar 2022

Gene writing helps develop advanced therapies safely and effectively

An international, multidisciplinary team of researchers from the Translational Synthetic Biology Laboratory at Pompeu Fabra University (Barcelona, Spain), led by Dr. Marc Güell, has published an article in the scientific journal Nature Communications.

14 Dec 2021

New gene therapy could help treat Duchenne muscular dystrophy

Researchers from UT Southwestern used a new kind of gene therapy to successfully treat mice with Duchenne muscular dystrophy (DMD), distinctively employing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein missing in several DMD patients.

3 May 2021

Duchenne Muscular Dystrophy News and Research

New Gene Therapy Approach Improves Muscle Strength in DMD Mice

Harnessing Enzymes for Making RNA Therapeutics

Review: Role of Alternative Splicing Defects in Major Neurodegenerative Diseases

Scarring on the Extracellular Matrix Causes Muscles to Stop Working in Duchenne Muscular Dystrophy

The relationship between cancer and DMD gene expression discovered

Scientists receive $2.5 million NIH grant to define the role of lipid droplets in muscle stem cell function

Cell-derived therapy may help treat ventricular arrhythmia

Cellular therapy leads to meaningful functional improvements in DMD patients

Gene writing helps develop advanced therapies safely and effectively

New gene therapy could help treat Duchenne muscular dystrophy

Revolutionary CRISPR-based genome editing system treatment destroys cancer cells

Study shows CRISPR/Cas9 system can effectively treat metastatic cancers

Study finds utrophin-based therapy could be used to treat Duchenne muscular dystrophy

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