This review evaluates the translatability of #CRISPR/Cas technology from a #NonClinical perspective, listing #ClinicalTrials and highlighting safety concerns in developing cellular & #GeneTherapy products. @cgtcatapult.bsky.social #medsky
#OpenAccess:
www.sciencedirect.com/science/arti...
#OpenAccess:
www.sciencedirect.com/science/arti...
September 21, 2025 at 5:56 PM
This review evaluates the translatability of #CRISPR/Cas technology from a #NonClinical perspective, listing #ClinicalTrials and highlighting safety concerns in developing cellular & #GeneTherapy products. @cgtcatapult.bsky.social #medsky
#OpenAccess:
www.sciencedirect.com/science/arti...
#OpenAccess:
www.sciencedirect.com/science/arti...
CRISPR/CasRx-mediated dual knockdown of Ctnnb1 and Smo effectively suppresses Wnt/β-catenin and Ihh signaling to reduce #osteoarthritis progression, highlighting a promising RNA-targeting #GeneTherapy strategy for OA treatment.
#OpenAccess: doi.org/10.1016/j.ge...
#OpenAccess: doi.org/10.1016/j.ge...
March 30, 2026 at 6:00 PM
CRISPR/CasRx-mediated dual knockdown of Ctnnb1 and Smo effectively suppresses Wnt/β-catenin and Ihh signaling to reduce #osteoarthritis progression, highlighting a promising RNA-targeting #GeneTherapy strategy for OA treatment.
#OpenAccess: doi.org/10.1016/j.ge...
#OpenAccess: doi.org/10.1016/j.ge...
A new review highlights the advances in DNA nucleases and delivery vectors for in vivo #GeneTherapy, outlining key developments, challenges, and their future potential to treat diverse #Diseases. #medsky
#OpenAccess: www.sciencedirect.com/science/arti...
#OpenAccess: www.sciencedirect.com/science/arti...
October 20, 2025 at 12:49 PM
A new review highlights the advances in DNA nucleases and delivery vectors for in vivo #GeneTherapy, outlining key developments, challenges, and their future potential to treat diverse #Diseases. #medsky
#OpenAccess: www.sciencedirect.com/science/arti...
#OpenAccess: www.sciencedirect.com/science/arti...
This study models PRPF6‑related #RetinitisPigmentosa using patient‑derived #RetinalOrganoids, showing that AAV‑mediated #GeneTherapy restores photoreceptor structure, rescues #ciliopathy defects, and reverses #RetinalDegeneration.
#OpenAccess: doi.org/10.1016/j.ge...
#OpenAccess: doi.org/10.1016/j.ge...
June 26, 2026 at 4:30 AM
This study models PRPF6‑related #RetinitisPigmentosa using patient‑derived #RetinalOrganoids, showing that AAV‑mediated #GeneTherapy restores photoreceptor structure, rescues #ciliopathy defects, and reverses #RetinalDegeneration.
#OpenAccess: doi.org/10.1016/j.ge...
#OpenAccess: doi.org/10.1016/j.ge...
This review highlights the role of #ProteinKinases in #NeurodegenerativeDiseases and explores kinase-targeted strategies—including inhibitors, #AntisenseOligonucleotides, and #GeneTherapy—for early intervention and disease management.
#OpenAccess in #STTT: doi.org/10.1038/s413...
#OpenAccess in #STTT: doi.org/10.1038/s413...
November 8, 2025 at 2:34 PM
This review highlights the role of #ProteinKinases in #NeurodegenerativeDiseases and explores kinase-targeted strategies—including inhibitors, #AntisenseOligonucleotides, and #GeneTherapy—for early intervention and disease management.
#OpenAccess in #STTT: doi.org/10.1038/s413...
#OpenAccess in #STTT: doi.org/10.1038/s413...
Une perspective thérapeutique fondée sur des cellules modifiées intégrant un ensemble de gènes fonctionnant en circuit synthétique à boucle fermée, afin de délivrer un effecteur thérapeutique en réponse à un signal pathologique déterminé.
bit.ly/4eUis7V
#OA #OpenAccess#GeneTherapy
#ENSdeLyon
bit.ly/4eUis7V
#OA #OpenAccess#GeneTherapy
#ENSdeLyon
July 25, 2024 at 9:27 AM
Une perspective thérapeutique fondée sur des cellules modifiées intégrant un ensemble de gènes fonctionnant en circuit synthétique à boucle fermée, afin de délivrer un effecteur thérapeutique en réponse à un signal pathologique déterminé.
bit.ly/4eUis7V
#OA #OpenAccess#GeneTherapy
#ENSdeLyon
bit.ly/4eUis7V
#OA #OpenAccess#GeneTherapy
#ENSdeLyon
This review comprehensively examines the structure and multifunctional roles of #p53, while highlighting emerging therapeutic strategies—including MDM2/4 inhibitors, mutant p53 reactivators, #GeneTherapy, and #Immunotherapy.
#OpenAccess: doi.org/10.1038/s413...
#OpenAccess: doi.org/10.1038/s413...
April 14, 2026 at 3:00 AM
This review comprehensively examines the structure and multifunctional roles of #p53, while highlighting emerging therapeutic strategies—including MDM2/4 inhibitors, mutant p53 reactivators, #GeneTherapy, and #Immunotherapy.
#OpenAccess: doi.org/10.1038/s413...
#OpenAccess: doi.org/10.1038/s413...
Researchers have developed a high-efficiency #AdenoviralVector system by using GAPDH to suppress #Adenovirus-induced #OxidativeStress, enabling rapid recombinant adenovirus production for #GeneTherapy.
#OpenAccess: doi.org/10.1016/j.ge...
#OpenAccess: doi.org/10.1016/j.ge...
February 17, 2026 at 3:02 PM
Researchers have developed a high-efficiency #AdenoviralVector system by using GAPDH to suppress #Adenovirus-induced #OxidativeStress, enabling rapid recombinant adenovirus production for #GeneTherapy.
#OpenAccess: doi.org/10.1016/j.ge...
#OpenAccess: doi.org/10.1016/j.ge...
In this review, researchers highlight in vivo #GeneTherapy as a promising approach for #Mutations, #DegenerativeDiseases, and #Tumors through precise #GenomeEditing and advanced delivery technologies.
#OpenAccess: doi.org/10.1016/j.ge...
#OpenAccess: doi.org/10.1016/j.ge...
May 28, 2026 at 2:54 PM
In this review, researchers highlight in vivo #GeneTherapy as a promising approach for #Mutations, #DegenerativeDiseases, and #Tumors through precise #GenomeEditing and advanced delivery technologies.
#OpenAccess: doi.org/10.1016/j.ge...
#OpenAccess: doi.org/10.1016/j.ge...
This review summarizes the molecular drivers of #Osteoarthritis and highlights innovative therapeutic strategies, including #GeneTherapy and stem cell interventions, to enhance joint regeneration and improve disease outcomes.
#STTT #OpenAccess: doi.org/10.1038/s413...
#STTT #OpenAccess: doi.org/10.1038/s413...
March 14, 2026 at 9:55 AM
This review summarizes the molecular drivers of #Osteoarthritis and highlights innovative therapeutic strategies, including #GeneTherapy and stem cell interventions, to enhance joint regeneration and improve disease outcomes.
#STTT #OpenAccess: doi.org/10.1038/s413...
#STTT #OpenAccess: doi.org/10.1038/s413...
This review summarizes the development of #DNA #Nucleases and delivery vectors, highlighting the progress and future potentials of in vivo #GeneTherapy for #GeneticDisorders, #DegenerativeDiseases, and #Cancers. #medsky
#OpenAccess:
www.sciencedirect.com/science/arti...
#OpenAccess:
www.sciencedirect.com/science/arti...
September 27, 2025 at 5:56 PM
This review summarizes the development of #DNA #Nucleases and delivery vectors, highlighting the progress and future potentials of in vivo #GeneTherapy for #GeneticDisorders, #DegenerativeDiseases, and #Cancers. #medsky
#OpenAccess:
www.sciencedirect.com/science/arti...
#OpenAccess:
www.sciencedirect.com/science/arti...
This review highlights #miRNAs as key regulators of #apoptosis in #BreastCancer, influencing radio- and #chemosensitivity, and underscores their potential as #GeneTherapy targets to overcome treatment resistance and improve therapeutic outcomes.
#OpenAccess: doi.org/10.1016/j.ge...
#OpenAccess: doi.org/10.1016/j.ge...
April 28, 2026 at 4:30 PM
This review highlights #miRNAs as key regulators of #apoptosis in #BreastCancer, influencing radio- and #chemosensitivity, and underscores their potential as #GeneTherapy targets to overcome treatment resistance and improve therapeutic outcomes.
#OpenAccess: doi.org/10.1016/j.ge...
#OpenAccess: doi.org/10.1016/j.ge...
Using #KidneyOrganoids, this #STTT study shows that AAV transduction during #GeneTherapy promotes #Nephrotoxicity by inducing #Genotoxicity and IL-1β secretion, which activates the canonical NFκB pathway. #medsky @harvardmed.bsky.social
#OpenAccess: doi.org/10.1038/s413...
#OpenAccess: doi.org/10.1038/s413...
October 15, 2025 at 9:30 AM
Using #KidneyOrganoids, this #STTT study shows that AAV transduction during #GeneTherapy promotes #Nephrotoxicity by inducing #Genotoxicity and IL-1β secretion, which activates the canonical NFκB pathway. #medsky @harvardmed.bsky.social
#OpenAccess: doi.org/10.1038/s413...
#OpenAccess: doi.org/10.1038/s413...
This #MostRead review discusses the applications & challenges of using #AAV8 as a vector for #GeneTherapy, highlighting the biological properties that make it a promising candidate vector for efficient #GeneTransfection. #medsky
#OpenAccess:
www.sciencedirect.com/science/arti...
#OpenAccess:
www.sciencedirect.com/science/arti...
June 18, 2025 at 5:56 PM
This #MostRead review discusses the applications & challenges of using #AAV8 as a vector for #GeneTherapy, highlighting the biological properties that make it a promising candidate vector for efficient #GeneTransfection. #medsky
#OpenAccess:
www.sciencedirect.com/science/arti...
#OpenAccess:
www.sciencedirect.com/science/arti...
A preclinical evaluation on the efficacy and safety of SIN-EFS-IL2RG.co vector- a self-inactivating lentiviral vector (SIN-LV) carrying IL2RG to potentiate #GeneTherapy in #XSCID. @UCLchildhealth #medsky
#OpenAccess: https://buff.ly/4g6Lqkc
#OpenAccess: https://buff.ly/4g6Lqkc
January 20, 2025 at 1:00 PM
A preclinical evaluation on the efficacy and safety of SIN-EFS-IL2RG.co vector- a self-inactivating lentiviral vector (SIN-LV) carrying IL2RG to potentiate #GeneTherapy in #XSCID. @UCLchildhealth #medsky
#OpenAccess: https://buff.ly/4g6Lqkc
#OpenAccess: https://buff.ly/4g6Lqkc
This review explores vector innovations, curative targets, and delivery strategies, while addressing obstacles in clinical translation and prospects for treating #neurologic disorders. #medsky #Neurology #GeneTherapy
#BIOI #OpenAccess: doi.org/10.15212/bio...
#BIOI #OpenAccess: doi.org/10.15212/bio...
September 24, 2026 at 2:14 PM
This review explores vector innovations, curative targets, and delivery strategies, while addressing obstacles in clinical translation and prospects for treating #neurologic disorders. #medsky #Neurology #GeneTherapy
#BIOI #OpenAccess: doi.org/10.15212/bio...
#BIOI #OpenAccess: doi.org/10.15212/bio...
#Biologics are broadening therapeutic strategies for cardiovascular disease.
This review highlights advances in #CardiacRegeneration, reverse remodeling, #GeneTherapy, vascular function and lipid modulation.
#STTT #OpenAccess: doi.org/10.1038/s413...
This review highlights advances in #CardiacRegeneration, reverse remodeling, #GeneTherapy, vascular function and lipid modulation.
#STTT #OpenAccess: doi.org/10.1038/s413...
September 12, 2026 at 11:30 AM
#Biologics are broadening therapeutic strategies for cardiovascular disease.
This review highlights advances in #CardiacRegeneration, reverse remodeling, #GeneTherapy, vascular function and lipid modulation.
#STTT #OpenAccess: doi.org/10.1038/s413...
This review highlights advances in #CardiacRegeneration, reverse remodeling, #GeneTherapy, vascular function and lipid modulation.
#STTT #OpenAccess: doi.org/10.1038/s413...
Can we reverse cardiac aging?
A longevity-linked BPIFB4 gene variant prevents diastolic dysfunction in #Progeria mice. This finding offers a promising #GeneTherapy strategy to combat #CardiovascularAging. @bristoluni.bsky.social
#STTT #OpenAccess: doi.org/10.1038/s413...
A longevity-linked BPIFB4 gene variant prevents diastolic dysfunction in #Progeria mice. This finding offers a promising #GeneTherapy strategy to combat #CardiovascularAging. @bristoluni.bsky.social
#STTT #OpenAccess: doi.org/10.1038/s413...
August 22, 2026 at 4:15 PM
Can we reverse cardiac aging?
A longevity-linked BPIFB4 gene variant prevents diastolic dysfunction in #Progeria mice. This finding offers a promising #GeneTherapy strategy to combat #CardiovascularAging. @bristoluni.bsky.social
#STTT #OpenAccess: doi.org/10.1038/s413...
A longevity-linked BPIFB4 gene variant prevents diastolic dysfunction in #Progeria mice. This finding offers a promising #GeneTherapy strategy to combat #CardiovascularAging. @bristoluni.bsky.social
#STTT #OpenAccess: doi.org/10.1038/s413...
Can activity-responsive #GeneTherapy prevent #Epilepsy? This study shows that an activity-dependent #Antioxidant strategy limits #OxidativeStress, protects neural circuits, and suppresses #Epileptogenesis in preclinical models.
#STTT #OpenAccess: doi.org/10.1038/s413...
#STTT #OpenAccess: doi.org/10.1038/s413...
August 4, 2026 at 2:45 AM
Can activity-responsive #GeneTherapy prevent #Epilepsy? This study shows that an activity-dependent #Antioxidant strategy limits #OxidativeStress, protects neural circuits, and suppresses #Epileptogenesis in preclinical models.
#STTT #OpenAccess: doi.org/10.1038/s413...
#STTT #OpenAccess: doi.org/10.1038/s413...
Researchers engineered a suppressor tRNA to restore Kir7.1 function and improve retinal physiology in a mouse model of #LeberCongenitalAmaurosis, demonstrating a promising #GeneTherapy strategy for inherited blindness. @uwmadison.bsky.social
#STTT #OpenAccess: doi.org/10.1038/s413...
#STTT #OpenAccess: doi.org/10.1038/s413...
July 28, 2026 at 4:10 PM
Researchers engineered a suppressor tRNA to restore Kir7.1 function and improve retinal physiology in a mouse model of #LeberCongenitalAmaurosis, demonstrating a promising #GeneTherapy strategy for inherited blindness. @uwmadison.bsky.social
#STTT #OpenAccess: doi.org/10.1038/s413...
#STTT #OpenAccess: doi.org/10.1038/s413...
This study presents an activity-dependent antioxidant #GeneTherapy approach that effectively reduces #OxidativeStress, neuronal damage, and seizure susceptibility while preventing #Epileptogenesis in preclinical #Epilepsy models.
#STTT #OpenAccess: doi.org/10.1038/s413...
#STTT #OpenAccess: doi.org/10.1038/s413...
June 25, 2026 at 6:01 AM
This study presents an activity-dependent antioxidant #GeneTherapy approach that effectively reduces #OxidativeStress, neuronal damage, and seizure susceptibility while preventing #Epileptogenesis in preclinical #Epilepsy models.
#STTT #OpenAccess: doi.org/10.1038/s413...
#STTT #OpenAccess: doi.org/10.1038/s413...
This study shows that #AAV8-mediated FVIII #GeneTherapy with #Tacrolimus–#Glucocorticoid prophylaxis achieves sustained FVIII activity and reduces bleeding in #HemophiliaA, with favorable safety outcomes.
#STTT #OpenAccess: doi.org/10.1038/s413...
#STTT #OpenAccess: doi.org/10.1038/s413...
April 6, 2026 at 6:16 PM
This study shows that #AAV8-mediated FVIII #GeneTherapy with #Tacrolimus–#Glucocorticoid prophylaxis achieves sustained FVIII activity and reduces bleeding in #HemophiliaA, with favorable safety outcomes.
#STTT #OpenAccess: doi.org/10.1038/s413...
#STTT #OpenAccess: doi.org/10.1038/s413...
This latest review highlights recent advances in understanding #m6Amethylation of RBM15, its molecular regulation in #Cancer and #MetabolicDiseases, and the emerging #therapeutic opportunities in future #GeneTherapy strategies. #medsky
#OpenAccess: www.sciencedirect.com/science/arti...
#OpenAccess: www.sciencedirect.com/science/arti...
November 21, 2025 at 3:53 PM
This latest review highlights recent advances in understanding #m6Amethylation of RBM15, its molecular regulation in #Cancer and #MetabolicDiseases, and the emerging #therapeutic opportunities in future #GeneTherapy strategies. #medsky
#OpenAccess: www.sciencedirect.com/science/arti...
#OpenAccess: www.sciencedirect.com/science/arti...
This new review highlights breakthroughs in in vivo #GeneTherapy, showcasing recent innovations, applications, challenges, and prospects, underscores its potential as a powerful tool to combat complex #HealthChallenges. #medsky
#OpenAccess:
www.sciencedirect.com/science/arti...
#OpenAccess:
www.sciencedirect.com/science/arti...
September 3, 2025 at 12:56 PM
This new review highlights breakthroughs in in vivo #GeneTherapy, showcasing recent innovations, applications, challenges, and prospects, underscores its potential as a powerful tool to combat complex #HealthChallenges. #medsky
#OpenAccess:
www.sciencedirect.com/science/arti...
#OpenAccess:
www.sciencedirect.com/science/arti...
A preclinical evaluation of the efficacy and safety of SIN-EFS-IL2RG.co vector, a self-inactivating lentiviral vector (SIN-LV) carrying #IL2RG, to potentiate GMP-compliant #GeneTherapy in #XSCID. #medsky
#MostRead #OpenAccess:
www.sciencedirect.com/science/arti...
#MostRead #OpenAccess:
www.sciencedirect.com/science/arti...
July 11, 2025 at 12:56 AM
A preclinical evaluation of the efficacy and safety of SIN-EFS-IL2RG.co vector, a self-inactivating lentiviral vector (SIN-LV) carrying #IL2RG, to potentiate GMP-compliant #GeneTherapy in #XSCID. #medsky
#MostRead #OpenAccess:
www.sciencedirect.com/science/arti...
#MostRead #OpenAccess:
www.sciencedirect.com/science/arti...