PMID- 35778330
OWN - NLM
STAT- MEDLINE
DCOM- 20220719
LR  - 20220921
IS  - 1558-1977 (Electronic)
IS  - 0889-8588 (Linking)
VI  - 36
IP  - 4
DP  - 2022 Aug
TI  - Adeno-Associated Virus Vector Design-Moving the Adeno-Associated Virus to a 
      Bioengineered Therapeutic Nanoparticle.
PG  - 667-685
LID - S0889-8588(22)00035-1 [pii]
LID - 10.1016/j.hoc.2022.04.002 [doi]
AB  - Although the number of market-approved gene therapies is still low, this new 
      class of therapeutics has become an integral part of modern medicine. The success 
      and safety of gene therapy depend on the vectors used to deliver the therapeutic 
      material. Adeno-associated virus (AAV) vectors have emerged as the most 
      frequently used delivery system for in vivo gene therapy. This success was 
      achieved with first-generation vectors, using capsids derived from natural AAV 
      serotypes. Their broad tropism, the high seroprevalence for many of the AAV 
      serotypes in the human population, and the high vector doses needed to transduce 
      a sufficient number of therapy-relevant target cells are challenges that are 
      addressed by engineering the capsid and the vector genome, improving the efficacy 
      of these biological nanoparticles.
CI  - Copyright (c) 2022 Elsevier Inc. All rights reserved.
FAU - Jaschke, Nico
AU  - Jaschke N
AD  - Institute of Experimental Hematology, Hannover Medical School, 
      Carl-Neuberg-Str.1, Hannover 30625, Germany.
FAU - Buning, Hildegard
AU  - Buning H
AD  - Institute of Experimental Hematology, Hannover Medical School, 
      Carl-Neuberg-Str.1, Hannover 30625, Germany; REBIRTH Research Center for 
      Translational Regenerative Medicine, Hannover Medical School, Carl-Neuberg-Str.1, 
      Hannover 30625, Germany; German Center for Infection Research, Partner Site 
      Hannover-Braunschweig. Electronic address: buening.hildegard@mh-hannover.de.
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PT  - Review
DEP - 20220628
PL  - United States
TA  - Hematol Oncol Clin North Am
JT  - Hematology/oncology clinics of North America
JID - 8709473
SB  - IM
MH  - Capsid
MH  - *Dependovirus/genetics
MH  - Genetic Vectors/genetics
MH  - Humans
MH  - *Nanoparticles
MH  - Seroepidemiologic Studies
OTO - NOTNLM
OT  - Adeno-associated virus (AAV) vectors
OT  - Capsid engineering
OT  - Gene therapy
OT  - Gene transfer
OT  - Vector genome engineering
COIS- Disclosures The authors declare no relevant conflicts of interest.
EDAT- 2022/07/02 06:00
MHDA- 2022/07/20 06:00
CRDT- 2022/07/01 22:14
PHST- 2022/07/02 06:00 [pubmed]
PHST- 2022/07/20 06:00 [medline]
PHST- 2022/07/01 22:14 [entrez]
AID - S0889-8588(22)00035-1 [pii]
AID - 10.1016/j.hoc.2022.04.002 [doi]
PST - ppublish
SO  - Hematol Oncol Clin North Am. 2022 Aug;36(4):667-685. doi: 
      10.1016/j.hoc.2022.04.002. Epub 2022 Jun 28.