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130 results about "Duchenne muscular dystrophy" patented technology

An inherited disorder characterized by progressive muscular weakness.

Exon skipping compositions for treating muscular dystrophy

InactiveUS20140315977A1Enhance the activity, cellular distribution, or cellular uptake of the antisense oligonucleotideOrganic active ingredientsSplicing alterationDuchenne muscular dystrophyMuscular dystrophy
Owner:SAREPTA THERAPEUTICS INC

Exon skipping compositions for treating muscular dystrophy

InactiveUS20150361428A1Enhance the activity, cellular distribution, or cellular uptake of the antisense oligonucleotideSplicing alterationSugar derivativesDuchenne muscular dystrophyMuscular dystrophy
Owner:SAREPTA THERAPEUTICS INC

Oligomers

Certain disclosed oligomers induce exon skipping during processing of myostatin pre-mRNA. The oligomers may be in a vector or encoded by the vector. The vector is used for inducing exon skipping during processing of myostatin pre-mRNA. A therapeutically effective amount of the oligomer may be administered to a subject patient such that exon skipping during processing of myostatin pre-mRNA is induced. The administration to a subject may be used in order to increase or maintain muscle mass, or slowing degeneration of muscle mass in the subject. The administration to a subject may ameliorate muscle wasting conditions, such as muscular dystrophy. Examples of such muscular dystrophies which may be so treated include Becker's muscular dystrophy, congenital muscular dystrophy, Duchenne muscular dystrophy, distal muscular dystrophy, Emery-Dreifuss muscular dystrophy, facioscapulohumeral muscular dystrophy (FSHD), limb-girdle muscular dystrophy, myotonic muscular dystrophy, and oculopharyngeal muscular dystrophy
Owner:ROYAL HOLLOWAY & BEDFORD NEW COLLEGE

Medicine for treating muscular dystrophy and myasthenia gravis, and its prepn. method

A Chinese medicine for treating the myophagism and myasthenia gravis caused by motor neuron diseases, prograssive myodystrophy and congenital myopathy is prepared from ginseng and epimedium. Its preparing process is also disclosed.
Owner:SHIJIAZHUANG YILING PHARMA

Methods of diagnosis and prognosis for a muscular dystrophy

The invention relates to the treatment, diagnosis, and prognosis of a muscular dystrophy or myopathy. The present inventors have found that the quantity of mu-crystallin is increased in a muscular dystrophy. In particular, the inventors have found that mu-crystallin is increased in facioscapulohumeral muscular dystrophy (FSHD). Based on the inventors' findings, the invention provides a novel means for the treatment, diagnosis, and prognosis of a muscular dystrophy or myopathy.
Owner:UNIV OF MARYLAND

DMD (Duchenne muscular dystrophy) gene capture probe and application thereof in DMD gene mutation detection

The invention discloses a DMD (Duchenne muscular dystrophy) gene capture probe and application thereof in DMD gene mutation detection. A preparation method of the DMD gene capture probe disclosed herein comprises: preparing N sub-probes, connecting the sub-probes to obtain the DMD gene capture probe; N is a natural number greater than or equal to 2; the N sub-probes can cover whole sequences of DMD gene; any two adjoining sub-probes on the DMD gene meet the case where there is one or more downstream nucleotide to the corresponding upstream sub-probe, which overlap with the upstream of the corresponding downstream sub-probe; the length of each of the N sub-probes is 50-150 bp. By detecting the DMD gene with the DMD gene capture probe, it is possible to detect whether the DMD gene experiences amplification mutation deletion / repetition or not, and it is also possible to precisely locate a break point region and the size of fragments and to detect DMD gene site mutations of a sample under detection.
Owner:MYGENOSTICS (CHONGQING) GENE TECH CO LTD

Pharmaco-cellular therapeutic method for the treatment of muscular dystrophies

A method of treating a muscular dystrophy disease in a patient includes administering an effective amount of a botanical drug isolated from Andrographis paniculata in combination with cell therapy. The method improves skeletal muscle performance.
Owner:PONTIFISIA UNIVERSIDAD KATOLIKA DE CHILE

Retinal dystrophin transgene and methods of use thereof

InactiveUS20080044393A1Alleviation of muscular dystrophy symptomReduce the possibilityBiocideNervous disorderBehavioral studyTransgene
Duchenne muscular dystrophy (DMD) is a progressive muscle disease that is caused by severe defects in the dystrophin gene and results in the patient's death by the third decade. The present invention utilizes the Double Mutant mice (DM) as an appropriate human model for DMD as these mice are deficient for both dystrophin and utrophin (mdx / +, utrn − / −), die at 3 months of age and suffer from severe muscle weakness, pronounced growth retardation, kyphosis, weight loss, slack posture, and immobility. Expression from a transgene of novel human retinal dystrophin Dp260 was shown to prevent premature death and reduce the severe muscular dystrophy phenotype to a mild clinical myopathy. Electromyography, histology, radiography, magnetic resonance imaging, and behavior studies concluded that DM transgenic mice grew normally, had normal spinal curvature and mobility, and had reduced muscle pathology. EMG and histologic data from transgenic DM mice showed decreased abnormalities to levels typical of mild myopathy, while the DM mice exhibited severe abnormalities commonly seen in human dystrophinopathies. The transgenic DM mice also had measurable movement levels comparable to those of untreated mdx mice and controls.
Owner:WHITE ROBERT L +2

Method and kit for detecting Duchenne muscular dystrophy gene defects, and primer composition

The invention relates to a method and a kit for detecting Duchenne muscular dystrophy gene defects, and a primer composition. For the Duchenne muscular dystrophy gene defects, a pre-embryo implantation genetic detection method which is strong in universality, high in diagnosis rate and low in cost is established in a second-generation sequencing platform. According to the method, multiple PCR is carried out on a detected sample for a coding region locus and a target SNP locus of a Duchenne muscular dystrophy gene, and a library is built; then a multiplex PCR product is subjected to high-throughput sequencing analysis, and a haplotype of a mutant allele is constructed by combining with family information, so that a genotype of an embryo is judged; and finally a Duchenne muscular dystrophy gene defect detection result of an offspring is determined. According to the scheme, a cell level pre-experiment is omitted; and based on a high-throughput sequencing technology, different label sequences can be added to samples, so that a large number of samples can be analyzed at one time, and the average sequencing depth can reach more than 100X.
Owner:REPRODUCTIVE & GENETIC HOSPITAL OF CITIC XIANGYA CO LTD +1

Treatment of muscular dystrophies and associated conditions by administration of monoamine oxidase inhibitors

Administration of a monoamine oxidase inhibitor is useful in the prevention and treatment of muscle dystrophy. Methods and compositions for inhibiting the production of reactive oxygen species in a muscle cell overproducing reactive oxygen species are provided herein.
Owner:UNIV DEGLI STUDI DI PADOVA

Kit for detecting mutation of duchenne muscular dystrophy causing gene and use thereof

The invention discloses a kit for detecting if a duchenne muscular dystrophy (DMD) causing gene has a mutation. The kit comprises the following main components: (1) 79 pairs of primers for PCR amplification and sequencing of 1 to 79 extrons of the DMD causing gene; (2) a polymerase chain reaction (PCR) amplification reagent; (3) a PCR product purification reagent; and (4) a DNA sequencing reagent. The kit disclosed by the invention is used for detecting the mutation of a duchenne muscular dystrophy causing gene, can quickly and conveniently detect people or patient with the DMD causing gene, can be used for prenatal diagnosis, prevention of birth of affected children, and can lower the incidence rate of the DMD.
Owner:上海佰真生物科技有限公司 +1

Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy

Disclosed herein are vectors that targets a dystrophin gene, encoding at least one Cas9 molecule or a Cas9 fusion protein, and at least one gRNA molecule (e.g., two gRNA molecules), and compositions and cells comprising such vectors. Also provided are methods for using the vectors, compositions and cells for genome engineering (e.g., correcting a mutant dystrophin gene), and for treating DMD.
Owner:EDITAS MEDICINE +1

Branched Chain Amino Acid Chelate

Compositions are disclosed comprising mineral / branched-chain amino acid chelates such as, for example, calcium leucinate, for increasing absorption of the branched-amino acid in a human or animal subject. Methods are also provided for using the compositions to increase branched-chain amino acid absorption to increase or maintain muscle mass in athletes or in individuals having diseases such as ALS, muscular dystrophy, sarcopenia associated with aging, or muscle atrophy associated with spinal cord injury, as well as to decrease mental fatigue.
Owner:BASTIAN ERIC D +1

Detection kit and method for Duchenne/Becker muscular dystrophy based on multiplex PCR (Polymerase Chain Reaction) trapping technique

The invention discloses a multiplex PCR (Polymerase Chain Reaction) trapping primer group for a DMD (Duchenne Muscular Dystrophy) gene, a mutation detection kit for the DMD gene and a mutation detection method for the DMD gene. The inventors provide the multiplex PCR trapping primer aiming at the sequences of 79 exons and bilateral 50bp introns and 23 known intron pathogenic mutations of the DMD gene; moreover, the mutation detection method of the DMD gene is provided based on the primer group; the mutation detection method is high in detection sensitivity, good in accuracy, highly consistentin amplification efficiency and low in requirement on quality of a DNA (Deoxyribonucleic Acid) sample, and is used for solving the problem that the complete detection on the mutation information, particularly on loss of heterozygosity / repetition, of the DMD gene is difficultly carried out in one time by adopting a multiplex PCR trapping technique in the prior art.
Owner:CAPITALBIO GENOMICS

Piperazine derivatives as trpml modulators

The new piperazine derivatives are modulators of TRPML and are useful in treating disorders related to TRPML activities such as lysosome storage diseases, muscular dystrophy, age-related common neurodegenerative diseases, oxidative stress or reactive oxygen species (ROS) related diseases, and ageing.
Owner:LYSOWAY THERAPEUTICS INC

Amplifying system and reagent kit for detecting DMD (Duchenne muscular dystrophy) gene exon copy number variation

The invention relates to an amplifying system for detecting human DMD (Duchenne muscular dystrophy) gene exon copy number variation. The amplifying system comprises an amplifying composition corresponding to the detection of 10 groups of exon copy number variation, wherein the amplifying composition comprises oligonucleotide sequences as shown from SEQID No.1 to SEQID No.36; the 10 exon purpose fragments including human DMD gene exons 4,8,17,44,45,47,48,50,51,52 are amplified through a real-time multiple fluorescent quantitation PCR reaction; and according to the detection result, whether thehuman DMD gene exon is subjected to deletion mutation or repeat mutation is judged. The invention also discloses a reagent kit for detecting human DMD (Duchenne muscular dystrophy) gene exon copy number variation. The amplifying system disclosed by the invention has the beneficial effects that through the detection result, the distinguishing of DMD carriers from DMD patients and normal people canbe realized, the result is reliable, the repeatability is good, the operation is easy, the equipment requirement is low, and the reagent cost is low; and the amplifying system can cover about 90% of copy number variation in the DMD carriers or the DMD patients, so that good balance between the detection cover degree and the detection exon number is achieved.
Owner:北京华瑞康源生物科技发展有限公司

Composition and Methods for the Treatment of Duchene Muscular Dystrophy

Patients are treated for muscular dystrophy by artificially increasing effective sarcospan activity in the patient's muscle tissue. Sarcospan (or fragments thereof) can be administered directly to the tissue, or their production can be induced by gene therapy. Modifications are also contemplated to be effective, including for example, a lipid, TAT or other tag. Also contemplated are compositions that include a protein, mRNA or gene stabilizer, degradation inhibitor, and / or oligomerization inhibitor. Increased presence of sarcospan can also be accomplished indirectly, using a vector having an entire sarcospan gene sequence, fragment or related construct, modulating a transcription factor, and so forth.
Owner:RGT UNIV OF CALIFORNIA

Application of salidroside in preparing medicine for treating duchenne muscular dystrophy

The invention relates to application of salidroside in preparing medicine for treating duchenne muscular dystrophy. According to the invention, experimental results show that salidroside can restrain skeletal muscle myolemma from rupture and necrosis, alleviate inflammatory reaction and fibrosis of skeletal muscles and enhance kinetism of skeletal muscles during the animal model paroxysm process of duchenne muscular dystrophy so as to express the function for treating duchenne muscular dystrophy.
Owner:SCI RES TRAINING CENT FOR CHINESE ASTRONAUTS
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