Developing AAV-based gene therapy for adrenoleukodystrophy (X-ALD)
Sign Up to like & getrecommendations! Published in 2019 at "Cytotherapy"
DOI: 10.1016/j.jcyt.2019.04.046
Abstract: Background & Aim Adrenoleukodystrophy (X-ALD) is an X-linked disease. ALD is caused by mutations of the gene encoding ATP-Binding cassette subfamily D member 1 (ABCD1), a peroxisomal transmembrane protein. Dysfunctional ABCD1 protein results in excessive… read more here.
Keywords: therapy; ald; based gene; aav based ... See more keywords
Emerging Thrombotic Disorders Associated with Virus-Based Innovative Therapies: From VITT to AAV Gene Therapy–Related Thrombotic Microangiopathy
Sign Up to like & getrecommendations! Published in 2024 at "Thrombosis and Haemostasis"
DOI: 10.1055/a-2413-4345
Abstract: Abstract Gene therapy is a promising therapeutic approach for treating life-threatening disorders. Despite the clinical improvements observed with gene therapy, immune responses either innate or adaptive against the vector used for gene delivery, can affect… read more here.
Keywords: aav based; associated virus; gene therapy; gene ... See more keywords
AAV-based gene therapies for the muscular dystrophies.
Sign Up to like & getrecommendations! Published in 2019 at "Human molecular genetics"
DOI: 10.1093/hmg/ddz128
Abstract: Muscular dystrophy is a group of progressive genetic diseases affecting the musculature which are characterized by inflammatory infiltrates, necrosis and connective tissue and fat replacement of the affected muscles. Unfortunately, treatments do not exist for… read more here.
Keywords: muscular dystrophies; muscular dystrophy; dystrophy; based gene ... See more keywords
Enhancing AAV-Based Industrial Systems With Cognitive IoT: Detecting AI-Manipulated Visual Data Using Graph-Based Methods
Sign Up to like & getrecommendations! Published in 2025 at "IEEE Internet of Things Journal"
DOI: 10.1109/jiot.2025.3550125
Abstract: The integration of Cognitive Internet of Things (IoT) sensors with autonomous aerial vehicles (AAVs) has transformed industrial sectors, such as monitoring, logistics, and infrastructure inspection. However, the advancement of visual synthesis technologies like generative adversarial… read more here.
Keywords: industrial systems; enhancing aav; aav based; graph based ... See more keywords
AAV-Based Gene Therapy: Opportunities, Risks, and Scale-Up Strategies
Sign Up to like & getrecommendations! Published in 2025 at "International Journal of Molecular Sciences"
DOI: 10.3390/ijms26178282
Abstract: Currently, the development of adeno-associated virus (AAV)-based gene therapy is a promising method for treating various diseases and is gaining increasing popularity. However, the use of AAV has certain drawbacks and faces limitations such as… read more here.
Keywords: therapy; aav based; based gene; therapy opportunities ... See more keywords