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Study demonstrates intricate relationship between Prpf19 and Exoc7 in inherited ataxias


Study demonstrates intricate relationship between Prpf19 and Exoc7 in inherited ataxias
Spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph Disease (MJD), describes the most common form of dominantly inherited ataxia in many populations worldwide, including Hong Kong and mainland China. SCA3 or MJD patients often present problems with gait and balance, blurred vision, and speech difficulties. The symptoms are progressive, and patients may eventually decline into a bed-ridden state. To date, SCA3 or MJD remains an incurable disorder.
Collaborating with the University of Oxford, Professor Ho Yin Edwin Chan s research team from the School of Life Sciences of The Chinese University of Hong Kong (CUHK) recently unveiled the counteracting relationship between pre-mRNA-processing factor 19 (Prpf19) and exocyst complex component 7 (Exoc7) in controlling the degradation of disease protein and neurodegeneration of the rare hereditary ataxia. The research findings have ....

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Retrotope Granted Rare Pediatric Disease Designation from FDA for Lead Development Candidate, RT001, in Two Life-Threatening Neurodegenerative Indications


Retrotope Granted Rare Pediatric Disease Designation from FDA for Lead Development Candidate, RT001, in Two Life-Threatening Neurodegenerative Indications
February 25, 2021 07:00 ET
| Source:
Retrotope
Retrotope
Los Altos, California, UNITED STATES
Company Currently Conducting Late-Stage Clinical Trials in Both Infantile Neuroaxonal Dystrophy (INAD) and Friedreich’s Ataxia (FA)
RT001 also Granted Fast Track Designation by FDA in FA; Orphan Drug Designation by European Medicines Agency in INAD
LOS ALTOS, Calif., Feb. 25, 2021 (GLOBE NEWSWIRE) Retrotope, a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class therapies for degenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted two rare pediatric disease designations to RT001, the company’s lead development candidate. The first rare pediatric disease designation i ....

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In Vivo Probes for Detecting Inflammation Markers in Humans and Animals


In Vivo Probes for Detecting Inflammation Markers in Humans and Animals
Thought LeadersProf. Parastoo HashemiSenior Lecturer in Bioengineering, Imperial London College
Associate Professor of Chemistry, University of South Carolina
In this interview, News-Medical Life Sciences talks to Parry Hashemi about the research on in vivo probes for detecting inflammation markers in humans and animals that she will present at Pittcon.
What are some traditional methods for detecting inflammation markers in humans and animals?
Traditionally, a blood or plasma sample will be taken. There are certain proteins that indicate an inflammation; for example, pro-inflammatory cytokines. If the levels of these are raised that corresponds to inflammation. ....

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New collaborative project to study the complex roles of lipids in health, disease


New collaborative project to study the complex roles of lipids in health, disease
A University of Minnesota-Mayo Clinic partnership has joined with the Zooniverse citizen scientist platform and the Francis Crick Institute in London to leverage world-leading expertise in microscope imaging with the power of citizen science to study the relationship between structure and function in biology to better understand health and disease.
The Minnesota Microscopy Infrastructure Partnership, led by the Mayo Clinic, the University of Minnesota, and the University s Hormel Institute, serves the high-end optical and electron microscope needs of more than 400 research laboratories in Minnesota and surrounding states. The collaboration is supported by the State of Minnesota. ....

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