Adeno-associated Virus (AAV) versus Immune Response.
immune response
rAAV
rare diseases
Journal
Viruses
ISSN: 1999-4915
Titre abrégé: Viruses
Pays: Switzerland
ID NLM: 101509722
Informations de publication
Date de publication:
25 01 2019
25 01 2019
Historique:
received:
05
12
2018
revised:
09
01
2019
accepted:
16
01
2019
entrez:
30
1
2019
pubmed:
30
1
2019
medline:
21
6
2019
Statut:
epublish
Résumé
Decades ago, Friedmann and Roblin postulated several barriers to gene therapy, including tissue targeting, delivery across the blood⁻brain barrier (BBB), and host immune responses. These issues remain pertinent till today. Since then, several advances have been made in elucidating structures of adeno-associated virus (AAV) serotypes, antibody epitopes, and ways to modify antibody-binding sites. AAVs capsid has also been engineered to re-direct tissue tropism, reduce ubiquitination, and promote passage across the BBB. Furthermore, the use of high(er) dose recombinant AAV (rAAV) has been accompanied by a better understanding of immune responses in both experimental animals and early clinical trials, and novel work is being performed to modulate the immune response. While the immune responses to rAAV remains a major challenge in translating experimental drugs to approved medicine, and will likely require more than a single solution, we now better understand the hurdles to formulate and test experimental solutions to surmount them.
Identifiants
pubmed: 30691064
pii: v11020102
doi: 10.3390/v11020102
pmc: PMC6409805
pii:
doi:
Substances chimiques
Capsid Proteins
0
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Review
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : NHLBI NIH HHS
ID : P01 HL112761
Pays : United States
Déclaration de conflit d'intérêts
The authors declare a conflict of interest. R. Jude Samulski is the founder and a shareholder at Asklepios BioPharmaceutical and Bamboo Therapeutics, Inc. He holds patents that have been licensed by UNC to Asklepios Biopharmaceutical, for which he receives royalties. He has consulted for Baxter Healthcare and has received payment for speaking.
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