Divergene

Researchers at the University of Wisconsin–Madison have devised a method using electric pulses to enhance the uptake of gene therapy material by liver cells, potentially reducing the dosage required for treatments like those for cystic fibrosis and diabetes. This breakthrough holds promise for improving the efficiency and affordability of gene therapies. #GeneTherapy #MedicalInnovation #HealthTech #Biotechnology #ResearchBreakthrough #Electroporation #LiverTreatment #MedicalScience #UniversityResearch #WisconsinMadison https://lnkd.in/gmfDWFHQ

A breakthrough gene therapy developed by scientists at Umeå University shows remarkable success in slowing the progression of amyotrophic lateral sclerosis (ALS) in a patient with an aggressive form of the disease caused by a mutation in the SOD1 gene. After four years of treatment, the patient maintains functional abilities such as climbing stairs and speaking, defying initial prognoses. This promising development underscores the potential of the therapy to significantly improve the lives of ALS patients, although further research is needed to optimize dosage and understand its broader applicability across different types of ALS. #ALSResearch #GeneTherapy #NeurologyBreakthrough #MedicalInnovation #RareDisease #HealthTech #Neuroscience #MedicalAdvancements #SOD1Mutation #ClinicalTrials https://lnkd.in/eHn-UVah

In a recent study, researchers have developed the SGMO Helper, a baculovirus-based insect cell production system for manufacturing adeno-associated viruses (AAV) used in gene and cell therapy. It offers enhanced flexibility in modulating Rep and Cap protein expression levels. Incorporating modifications to the AAV6 capsid sequence improves yield, capsid integrity, and potency, resulting in improved production compared to traditional methods like Bac-RepCap. Next-generation sequencing analysis confirms stability over six passages, and scalable production in bioreactors enhances yield and potency, making the SGMO Helper an advanced platform for AAV manufacturing, crucial for cutting-edge gene and cell therapies. #GeneTherapy #CellTherapy #AAVProduction #BioreactorScaleup #NextGenSequencing #InsectCellSystems #BaculovirusExpression #AAV6Improvements #TherapeuticViruses #MedicalResearch https://lnkd.in/eCSyZT2S

Familial dysautonomia (FD) is a rare neurodevelopmental and neurodegenerative disease stemming from a mutation in the ELP1 gene, causing visual impairment due to retinal ganglion cell (RGC) death. Two therapeutic approaches, gene replacement therapy and small molecule splicing modifiers, show promise in rescuing RGCs in mouse models, potentially paving the way for future treatments in FD patients. #FamilialDysautonomia #Neurodevelopmental #Neurodegenerative #GeneticDisorder #MedicalResearch #RareDisease #GeneTherapy #PreclinicalResearch #SplicingModifiers #ELP1Gene https://lnkd.in/eaMDVAJd

This study investigates the effectiveness of AAV6 as a gene therapy vector for joint diseases, highlighting concerns like low transduction efficacy and off-target expression. Researchers engineered novel AAV capsids, with AAV62 showing improved transduction efficiency over AAV6. AAV6D, with a specific deletion, demonstrated significantly higher transduction, suggesting potential for improved joint targeting and reduced off-target effects, indicating promise for future intra-articular gene therapy strategies. #GeneTherapy #AAVGeneTherapy #JointDiseases #AAV6 #AAVCapsids #IntraArticular #TransductionEfficiency #AAVMutants #Synoviocytes #Chondrocytes https://lnkd.in/ej2z46Gz

A recent study introduces two novel AAV capsids, Pep2hSC1 and Pep2hSC2, developed through functional transduction-RNA selection, demonstrating potent transgene delivery capabilities across various models. These breakthroughs hold potential for addressing disorders related to Schwann cells and peripheral nervous system injuries. #GeneTherapy #BiomedicalResearch #GeneticDisorders #AAVVectors #SchwannCells #ClinicalTrials #TransgeneDelivery #TherapeuticSolutions #IncurableDiseases #MedicalInnovation https://lnkd.in/eGZEXZUX

A study examined the potential for gene therapy in Friedreich’s ataxia, a disorder caused by mutations in the FXN gene leading to mitochondrial dysfunction. Researchers administered recombinant adeno-associated virus encoding human frataxin (AAVrh.10hFXN) in mice, inducing dose-dependent expression of mature human frataxin (hFXN) in the heart and liver, approaching endogenous mouse frataxin (mFXN) levels. While hFXN was found to be truncated, it was at different positions than mFXN, suggesting AAVrh.10hFXN as a promising approach for inducing therapeutic hFXN expression. #FriedreichsAtaxia #GeneTherapy #MitochondrialDysfunction #Frataxin #AAVrh10hFXN #NeurodegenerativeDisorder #RareDisease #ResearchUpdate #MedicalScience #TherapeuticIntervention https://lnkd.in/euXFhSXw

Epidemiological studies highlight the protective role of the APOE ε2 allele against Alzheimer's disease, showing later onset, milder symptoms, and reduced neuropathological effects compared to the ε4 allele. A gene therapy approach delivering APOE ε2 to the brain reduces Aβ plaque deposition, synaptic loss, and microglial activation in mice expressing the ε4 allele, suggesting a potential therapeutic avenue for Alzheimer's, particularly for ε4 carriers. This research underscores the promise of exogenous APOE ε2 in mitigating Alzheimer's pathology, even in individuals genetically predisposed with the ε4 allele. #AlzheimersResearch #GeneTherapy #Neuroscience #BrainHealth #APOE #DementiaAwareness #Neurodegeneration #HealthTech #MedicalBreakthrough #AlzheimersPrevention https://lnkd.in/e2xeFATw

Researchers at Duke University School of Medicine have enhanced humanized animal models, crucial for studying drug effects without human subjects, by modifying adeno-associated viruses (AAVs) delivery into human liver cells. Their method increases accuracy in modeling drug responses, potentially revolutionizing clinical trials by aiding in candidate selection and identifying issues prior to human testing. This advancement promises to expedite drug development, offering more effective treatments and better patient outcomes. #BiomedicalResearch #DrugDevelopment #ClinicalTrials #GeneTherapy #HumanizedModels #MedicalInnovation #HealthTech #PrecisionMedicine #HealthcareAdvancements #PatientOutcomes https://lnkd.in/eRbmp6vJ

This study explores a novel gene therapy method for treating heart failure by developing an endovascular technique for global left ventricular (LV) transduction. Utilizing domestic pigs, researchers achieved widespread LV myocardium coverage through retrograde injections into the three main LV vein branches, significantly improved by transient occlusions of corresponding coronary arteries and anastomotic veins. The method demonstrated efficient gene transduction using both adenovirus (Ad) and adeno-associated virus (AAV) vectors, suggesting promise for global LV gene transfer in cardiac gene therapy. #HeartHealth #GeneTherapy #CardiacCare #MedicalResearch #HealthTech #HeartDisease #InnovativeMedicine #Biotechnology #ClinicalTrials #HealthyLiving https://lnkd.in/eVHFw3Pt