Centre for Eye Research Australia (CERA)’s Post

Gene therapy is one of the most exciting developments in modern medicine, with massive potential to transform the lives of patients with genetic diseases. However, severe immune-mediated adverse events may limit the clinical utility of these therapies in some patients. Research is uncovering the role of complement factors as drivers of some of these adverse events, for example in thrombotic microangiopathy (TMA). Being able to measure complement activation close to real-time holds great promise to quickly detect these adverse events as they emerge, and potentially significantly improve patient safety. Find out how Kypha’s complement technology can help spot emergent adverse events in close to real-time. Visit https://lnkd.in/eZJ3ekqS

Gene therapy is one of the most exciting developments in modern medicine, with massive potential to transform the lives of patients with genetic diseases. However, severe immune-mediated adverse events may limit the clinical utility of these therapies in some patients. Research is uncovering the role of complement factors as drivers of some of these adverse events, for example in thrombotic microangiopathy (TMA). Being able to measure complement activation close to real-time holds great promise to quickly detect these adverse events as they emerge, and potentially significantly improve patient safety. Find out how Kypha’s complement technology can help spot emergent adverse events in close to real-time. Visit https://lnkd.in/eSi-c8v5

Ophthopedia Update:Corrigendum: The publisher would like to make the following correction to “Single center experience with Voretigene Neparvovec gene augmentation therapy in RPE65-mutation associated inherited retinal degeneration” (Ophthalmology 2024;2:161-178). The first sentence of the Results section in the Abstract should read as follows (correction in boldface): Results: Thirty eyes of 19 patients were analyzed (10 pediatric eyes < 20 years; 20 adult eyes > 20 years of age; overall range: 8-40 years) with a median follow-up of 15 months (range, 1-32). #Ophthalmology #ophthotwitter #eyecare

Don't miss the last #EJPRD #MOOC on #innovativetherapies and #personalizedmedicine for #rarediseases! In this course, we explore the most innovative approaches for developing therapies (such as #genetherapy, #celltherapy, protein-based approaches), the concepts of #regenerativemedicine, #tissueengineering and #transplants and how they can help the treatment of #rarediseases. We also introduce #personalizedmedicine, explain how it differs from the classical “one fits all approach" and discuss the current real-world challenges related to its development. The MOOC was developed by: Foundation For Rare Diseases, Consorzio per Valutazioni Biologiche e Farmacologiche (CVBF) and ERN TransplantChild.

The European Joint Programme on Rare Diseases #EJPRD is offering a new five-week online course to explore the latest therapeutic treatments being developed in medical research, such as: gene therapy, protein-based treatments, regenerative medicine, cell therapy and tissue engineering. more: https://lnkd.in/ezjU26pe The online course is part of a series of courses including: - Innovative Therapies and Personalised Medicine for Rare Diseases - Diagnosing Rare Diseases: from Clinic to Research and Back - From the lab to the clinic: Translational Research for Rare Diseases - Health Data Ethics & Regulatory Frameworks in Rare Disease Research

A recently published chapter of the Handbook of Clinical Neurology, highlights some of the associated complexities in “The health economics of cell and gene therapies”. ➡️ https://buff.ly/3XWH73X In this chapter, written by Laura Beswick, Head of Health Economics and Market Access at the Cell and Gene Therapy Catapult, the cost utility approach of economic evaluation is discussed, focusing on the considerations that occur more commonly in cell and gene therapies compared to conventional medicines, how these considerations create challenges in interpreting the evidence and coming to conclusions on value, and what tools exist for understanding the level of decision uncertainty. Read the chapter ☝️ #CellTherapy #GeneTherapy #AdvancedTherapies

This study enhances the understanding of the diverse effects of endometrial tissue cryopreservation on cellular characteristics, gene expression and epigenetic modifications such as histone modifications, DNA methylation and miRNA levels. The findings presented by Deimantė Žukauskaitė, Aistė Zentelytė, Erika Girniūtė, and Ruta Navakauskiene progress the potential for optimizing cryopreservation protocols and advancing the application of cryopreserved tissues across various fields, including regenerative medicine, cell therapy and reproductive health. Moreover, the findings of this study suggest the potential application of slow cooling cryopreservation not only for normal endometrial tissue samples, but also for pathological tissue samples with the purpose of isolating stromal cell cultures. Journal subscribers can read the full paper from our in-press articles section now https://lnkd.in/evSqw9bS   #ExperimentalReproductiveBiology # #Endometrium #Reproduction #Cryopreservation #Epigenetics #DnaMethylation #HistoneModification #miRNA

Neurofit is proud to have contributed to the research conducted by the Cognigenics team. Our involvement included administering a battery of behavioral tests, including the T-maze, Novel Object Recognition, and Light-Dark tests. The complete research findings are detailed in the publication and are further developed in an insightful article by Dr. Troy Rohn titled "Embracing a New Era in Medicine Through Gene Therapy." You can read the article at the following link: https://lnkd.in/eqxfHkDF

‘Autosomal recessive deafness 9, caused by mutations of the OTOF gene, is characterised by congenital or prelingual, severe-to-complete, bilateral hearing loss. However, no pharmacological treatment is currently available for congenital deafness. In this Article, we report the safety and efficacy of gene therapy with an adeno-associated virus (AAV) serotype 1 carrying a human OTOF transgene (AAV1-hOTOF) as a treatment for children with autosomal recessive deafness 9.’ https://lnkd.in/d4EY99gV

OSU: nonviral gene therapy relieves back pain and repairs damaged disc in mice https://lnkd.in/eAqtvsNe