Blood-brain barrier shuttle peptides enhance AAV transduction in the brain after systemic administration.

TL;DR: Genetically modifying AAV vectors to increase their transduction efficiency, vector tropism and ability to avoid the host immune response may further increase the success of AAV gene therapy.

TL;DR: In this paper , the authors provide an up-to-date and comprehensive guideline for researchers in diverse fields, offering perspectives on further development of brain-targeted drug delivery system, including passive transcytosis, intranasal administration, ligands conjugation, membrane coating and stimuli-triggered BBB disruption.

TL;DR: In this paper , different approaches to improve Adeno-associated virus (AAV) vectors for gene therapy with a focus on AAV capsid selection and engineering, strategies to overcome anti-AAV immune response, and vector genome design are discussed.

TL;DR: With these techniques more effective and even safer drugs can be developed for the treatment of devastating brain disorders, both technological and mechanistic tools are available to assist in overcoming the blood–brain barrier.

TL;DR: The current status, strengths, and weaknesses of peptides as medicines and the emerging new opportunities in peptide drug design and development are discussed.

TL;DR: In this article, a single portal vein infusion of a recombinant adeno-associated viral vector (rAAV) expressing canine Factor IX (F.IX) resulted in long-term expression of therapeutic levels of F.IX in dogs with severe hemophilia B.

TL;DR: Peripheral-vein infusion of scAAV2/8-LP1-hFIXco resulted in FIX transgene expression at levels sufficient to improve the bleeding phenotype, with few side effects.

TL;DR: It is shown that adeno-associated virus (AAV) 9 injected intravenously bypasses the BBB and efficiently targets cells of the central nervous system (CNS) and may enable the development of gene therapies for a range of neurodegenerative diseases.