Duchenne Muscular Dystrophy Drug Discovery Service
Creative Biolabs has done extensive research and completed multiple projects in the field of neuroscience. Based on our years of experience and advanced techniques, we are confident in offering a variety of services for our clients all over the world.
Introduction of Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a severe progressive muscle-wasting disease. This disease primarily affects boys and is much rarer in girls. Muscle weakness usually begins around age four and worsens rapidly. Till now, there is no known cure, while gene therapy as a potential treatment is in the early stages of research. With optimal medical care, most DMD patients die of heart failure and/or respiratory failure between the ages of 20 and 40.
Symptoms of Duchenne Muscular Dystrophy
- Muscle weakness
- Fatigue
- Difficulty with motor skills
- Falls
- Cardiomyopathy, especially dilated cardiomyopathy
- Occasional congestive heart failure or arrhythmia
Fig.1 Cross-sectional staining of healthy muscle and skeletal muscle from a patient with Duchenne muscular dystrophy. (Duan, 2021)
Cause of Duchenne Muscular Dystrophy
DMD is always caused by mutations in DMD that encode dystrophin. These mutations would prevent the generation of the muscle isoform of dystrophin (Dp427m) and also result in Becker muscular dystrophy (BMD). BMD is a subtype of DMD and generally presents as a milder form of muscular dystrophy. Converting the DMD type to the BMD type is the focus of research to improve symptoms. In DMD, frameshifting mutations and nonsense mutations would result in non-functional and unstable dystrophin. Studies have shown that thousands of different mutations can be detected in DMD. Among them, the proportion of deletion mutations is 60-70%, the proportion of repeat mutations is 20%, and the remaining 5-10% are point mutations, small deletions, or insertions.
Management of Duchenne Muscular Dystrophy
Despite significant therapeutic advances over the past few decades, DMD remains incurable. Nevertheless, based on the published best care guidelines, there are a series of multidisciplinary medical, surgical, and rehabilitative approaches to improve quality of life and longevity. The primary care modalities include respiratory care, cardiac care, orthopedic management, endocrinological management, gastrointestinal management, urological management, as well as neurodevelopmental and neuropsychological management. Currently, therapeutic development for DMD is primarily focused on restoring dystrophin production and reducing the secondary consequences of dystrophin deficiency. For example, stem cell transplantation can be used to improve muscle quality, while gene therapy aims to restore the missing dystrophin.
Duchenne Muscular Dystrophy Drug Discovery Service
Equipped with world-leading platforms and extensive experience, Creative Biolabs provides one-stop drug discovery service for our clients. From the basic target identification to the final IND-enabling, wee can meet all the needs of customers.
Creative Biolabs is a leading service provider that focuses on neuroscience research. We can assist you in designing the best research outline customized to meet the requirements of your programs. If you are interested in our services and products, please do not hesitate to contact us for more details.
Reference
- Duan, D.; et al. Duchenne muscular dystrophy. Nature Reviews Disease Primers. 2021, 7(1): 1-19.
- NeuroMab™ Anti-EPHB2 Antibody(NRP-0422-P1220) (Cat#: NRP-0422-P1220)
- NeuroMab™ Anti-GARP Antibody(NRP-0422-P1639) (Cat#: NRP-0422-P1639)
- NeuroMab™ Anti-TREM2 Antibody(NRP-0422-P792) (Cat#: NRP-0422-P792)
- iNeuMab™ Rabbit Anti-LRRK2 Monoclonal Antibody (CBP1887) (Cat#: NAB-08-PZ735)
- Mouse Anti-SCN5A Monoclonal Antibody (CBP708) (Cat#: NAB-0720-Z2720)
- NeuroMab™ Anti-SEZ6 Antibody(NRP-0422-P517) (Cat#: NRP-0422-P517)
- NeuroMab™ Anti-Tau Antibody(NRP-0422-P2275) (Cat#: NRP-0422-P2275)
- NeuroMab™ Anti-pTau Antibody(NRP-0422-P1719) (Cat#: NRP-0422-P1719)
- NeuroMab™ Anti-Amyloid Beta 1-15 Antibody(NRP-0422-P867) (Cat#: NRP-0422-P867)
- NeuroMab™ Anti-CD32b Antibody(NRP-0422-P1803) (Cat#: NRP-0422-P1803)
- Mouse Glioma Cell Line GL261-GFP (Cat#: NCL-2108P04)
- Human Astrocytes, Immortalized (Cat#: NCL-2105-P182-AM)
- Human Brain Astroblastoma U-87 MG (Cat#: NCL2110P117)
- Human Dental Pulp Stem Cells (Cat#: NRZP-1122-ZP113)
- iNeu™ Human Neural Stem Cell Line (Cat#: NCL200552ZP)
- iNeu™ Human Schwann Cell (Cat#: NCL-2103-P63)
- Mouse Glioma Cell Line GL261 (Cat#: NCL-2108P28)
- Human Astrocytes (Cat#: NCC20-9PZ01)
- Human Neurons Isolated from Cortex (Cat#: NCL-21P6-023)
- Rat Retinal Muller Cell Line, Immortalized (Cat#: NCL-21P6-192)
- Beta Amyloid (1-40), Aggregation Kit (Cat#: NRZP-0323-ZP199)
- Human Poly ADP ribose polymerase,PARP Assay Kit (Cat#: NRZP-1122-ZP62)
- Beta Amyloid (1-42), Aggregation Kit (Cat#: NRZP-0323-ZP200)
- Alpha Synuclein Aggregation Kit (Cat#: NRZP-1122-ZP15)
- Alpha-Synuclein Aggregation Assay Kit (Cat#: NRZP-1122-ZP37)
- Amyloid beta 1-42 Kit (Cat#: NRP-0322-P2170)
- Human GFAP ELISA Kit [Colorimetric] (Cat#: NPP2011ZP383)
- Human Tau Aggregation Kit (Cat#: NRP-0322-P2173)
- pAAV-syn-jGCaMP8m-WPRE (Cat#: NTA-2106-P062)
- pAAV-syn-jGCaMP8s-WPRE (Cat#: NTA-2106-P063)
- pAAV-syn-FLEX-jGCaMP8s-WPRE (Cat#: NTA-2106-P066)
- AAV2/9-hEF1a-fDIO-eNpHR 3.0-mCherry-WPRE-pA (Cat#: NTA-2012-ZP78)
- pAAV-syn-FLEX-jGCaMP8f-WPRE (Cat#: NTA-2106-P064)
- Dextran, Cy5 Labeled, 2000 kDa (Cat#: NRZP-0722-ZP22)
- AAV2 Full Capsids, Reference Standards (Cat#: NTC2101070CR)
- pAAV-syn-FLEX-jGCaMP8m-WPRE (Cat#: NTA-2106-P065)
- VSV-eGFP (Cat#: NTA-2011-ZP20)
- rAAV-E-SARE-Cre-ERT2-PEST-WPRE-hGH polyA (Cat#: NTA-2010-TT342)
- Human superoxide dismutase 3, extracellular (SOD3) (NM_003102) ORF clone, Untagged (Cat#: NEP-0521-R0808)
- App Rat amyloid beta (A4) precursor protein (App)(NM_019288) ORF clone, Untagged (Cat#: NEP-0421-R0053)
- ABCA1 Antisense Oligonucleotide (NV-2106-P27) (Cat#: NV-2106-P27)
- Tau Antisense Oligonucleotide (IONIS-MAPTRx) (Cat#: NV-2106-P29)
- Rat Parkinson disease (autosomal recessive, juvenile) 2, parkin (Park2) (NM_020093) ORF clone/lentiviral particle, Myc-DDK Tagged (Cat#: NEP-0621-R0041)
- Lenti of Human TAR DNA binding protein (TARDBP) (NM_007375) ORF clone, mGFP Tagged (Cat#: NEP-0521-R0832)
- Human presenilin 1 (PSEN1), transcript variant 2 (NM_007318) ORF clone, TurboGFP Tagged (Cat#: NEP-0421-R0140)
- Human huntingtin (HTT) (NM_002111) ORF clone, Myc-DDK Tagged (Cat#: NEP-0521-R0497)
- Mouse SOD1 shRNA Silencing Adenovirus (Cat#: NV-2106-P14)
- Human huntingtin-associated protein 1 (HAP1) transcript variant 2 (NM_177977) ORF clone, Myc-DDK Tagged (Cat#: NEP-0521-R0676)
- NeuroBiologics™ Monkey Cerebrospinal Fluid (Cat#: NRZP-0822-ZP495)
- NeuroBiologics™ Human Cerebrospinal Fluid (Cat#: NRZP-0822-ZP491)
- NeuroBiologics™ Mouse Cerebrospinal Fluid (Cat#: NRZP-0822-ZP497)
- NeuroBiologics™ Rat Cerebrospinal Fluid (Cat#: NRZP-0822-ZP496)
- NeuroBiologics™ Pig Cerebrospinal Fluid (Cat#: NRZP-0822-ZP498)
- NeuroPro™ Anti-PON1 BBB Shuttle Protein (Cat#: NRZP-0423-ZP507)
- NeuroPro™ Anti-NAGLU BBB Shuttle Protein (Cat#: NRZP-0423-ZP506)
- NeuroPro™ Anti-IDUA BBB Shuttle Protein (Cat#: NRZP-0423-ZP502)
- NeuroPro™ Anti-GDNF BBB Shuttle Protein (Cat#: NRZP-0423-ZP500)
- NeuroPro™ Anti-SGSH BBB Shuttle Protein (Cat#: NRZP-0423-ZP505)
- NeuroPro™ Anti-IDUA BBB Shuttle Protein (Cat#: NRZP-0423-ZP498)
- NeuroPro™ Anti-idursulfase BBB Shuttle Protein (Cat#: NRZP-0423-ZP497)
- NeuroPro™ Anti-TNFR BBB Shuttle Protein (Cat#: NRZP-0423-ZP510)
- NeuroPro™ Anti-IDS BBB Shuttle Protein (Cat#: NRZP-0423-ZP503)
- NeuroPro™ Anti-Erythropoietin BBB Shuttle Protein (Cat#: NRZP-0423-ZP499)