AAV vectors: The Rubik's cube of human gene therapy

A Pupo, A Fernández, SH Low, A François… - Molecular Therapy, 2022 - cell.com
Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …

Advances in therapeutic applications of extracellular vesicles

OPB Wiklander, MÁ Brennan, J Lötvall… - Science translational …, 2019 - science.org
Extracellular vesicles (EVs) are nanometer-sized, lipid membrane–enclosed vesicles
secreted by most, if not all, cells and contain lipids, proteins, and various nucleic acid …

Exosome-mediated delivery of gene vectors for gene therapy

L Duan, L Xu, X Xu, Z Qin, X Zhou, Y Xiao, Y Liang… - Nanoscale, 2021 - pubs.rsc.org
Gene vectors are nucleic acids that carry genetic materials or gene editing devices into cells
to exert the sustained production of therapeutic proteins or to correct erroneous genes of the …

[HTML][HTML] AAV vectors applied to the treatment of CNS disorders: Clinical status and challenges

L Kang, S Jin, J Wang, Z Lv, C Xin, C Tan… - Journal of Controlled …, 2023 - Elsevier
In recent years, adeno-associated virus (AAV) has become the most important vector for
central nervous system (CNS) gene therapy. AAV has already shown promising results in …

Methodological guidelines to study extracellular vesicles

FAW Coumans, AR Brisson, EI Buzas… - Circulation …, 2017 - Am Heart Assoc
Owing to the relationship between extracellular vesicles (EVs) and physiological and
pathological conditions, the interest in EVs is exponentially growing. EVs hold high hopes …

Deafness: from genetic architecture to gene therapy

C Petit, C Bonnet, S Safieddine - Nature Reviews Genetics, 2023 - nature.com
Progress in deciphering the genetic architecture of human sensorineural hearing
impairment (SNHI) or loss, and multidisciplinary studies of mouse models, have led to the …

Therapeutic and diagnostic translation of extracellular vesicles in cardiovascular diseases: roadmap to the clinic

S Sahoo, M Adamiak, P Mathiyalagan, F Kenneweg… - Circulation, 2021 - Am Heart Assoc
Exosomes are small membrane-bound vesicles of endocytic origin that are actively
secreted. The potential of exosomes as effective communicators of biological signaling in …

Immunogenicity of recombinant adeno-associated virus (AAV) vectors for gene transfer

M Arjomandnejad, I Dasgupta, TR Flotte, AM Keeler - BioDrugs, 2023 - Springer
Recombinant adeno-associated viruses (AAVs) have emerged as promising gene delivery
vehicles resulting in three US Food and Drug Administration (FDA) and one European …

Extracellular vesicles: Novel promising delivery systems for therapy of brain diseases

D Rufino-Ramos, PR Albuquerque, V Carmona… - Journal of Controlled …, 2017 - Elsevier
Extracellular vesicles (EVs) are cell-derived membrane vesicles virtually secreted by all
cells, including brain cells. EVs are a major term that includes apoptotic bodies …

[HTML][HTML] Efficient production and enhanced tumor delivery of engineered extracellular vesicles

DC Watson, D Bayik, A Srivatsan, C Bergamaschi… - Biomaterials, 2016 - Elsevier
Extracellular vesicles (EV), including exosomes and microvesicles, are nano-sized
intercellular communication vehicles that participate in a multitude of physiological …