Adeno-associated Virus Vectors in Gene Therapy

Mary, Bertin; Khan, Noureen; Arumugam, Sathyathithan; Saxena, Harshit; Kumar, Mohit; Manimaran, Paramasivam; Chattopadhyay, Sourav; Jayandharan, Giridhara R.

doi:10.1007/978-981-13-0481-1_2

Cited by 4 publications

(2 citation statements)

References 144 publications

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“…Metabolic disorders mainly affect a few organs, including the liver, intestine, and pancreas of the body. Among them, the liver is a preferable target organ for gene therapy, and many types of AAV, such as AAV8, AAV9, and AAV-DJ, have strong cytotropism for the liver [176,177]. Therefore, based on CRISPR-mediated gene editing, researchers have developed various gene therapy approaches for treating metabolic diseases of the liver, such as familial hypercholesterolemia (FH), hepatorenal tyrosinemia type 1 (HT1), phenylketonuria (PKU), and so on.…”

Section: Metabolic Disordersmentioning

confidence: 99%

CRISPR-Based Tools for Fighting Rare Diseases

Gao

Wang

2022

Life

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Rare diseases affect the life of a tremendous number of people globally. The CRISPR-Cas system emerged as a powerful genome engineering tool and has facilitated the comprehension of the mechanism and development of therapies for rare diseases. This review focuses on current efforts to develop the CRISPR-based toolbox for various rare disease therapy applications and compares the pros and cons of different tools and delivery methods. We further discuss the therapeutic applications of CRISPR-based tools for fighting different rare diseases.

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Section: Metabolic Disordersmentioning

confidence: 99%

CRISPR-Based Tools for Fighting Rare Diseases

Gao

Wang

2022

Life

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“…To date, 13 different AAV serotypes (AAV1 to AAV13) have been identified based on the phylogenetic classification, and more than 100 variants from human and primate origin are known. Variants differ in their capsid structures and display variable transduction efficiency, tissue-or cell-type tropism, and immunological profile [7][8][9]. Efforts have been made to isolate or engineer new AAV serotypes [10][11][12][13].…”

Section: Introductionmentioning

confidence: 99%

Popularizing Recombinant Baculovirus-derived OneBac System for Laboratory Production of all Recombinant Adeno-associated Virus Vector Serotypes

Han

Duan

et al. 2021

CGT

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Objective: On the basis of our previously established single recombinant baculovirus expression vector (BEV)-derived OneBac system, we have optimized the process and expanded the rAAV production range to the full range of serotypes rAAV1-13. Firstly, the AAV Cap gene was optimized to translate by ribosome leaky scanning and the gene of interest (GOI) was cloned into the pFD/Cap-(ITR-GOI)-Rep2 shutte plasmid. Following the classical Bac-to-Bac method, sufficient BEV stock containing all rAAV packaging elements can be quickly obtained. Finally, we can repeatedly scale up production of rAAVs in one week by using a single BEV to infect suspension-cultured Sf9 cells. The rAAV1-13 show relatively high yields ranging from 5×104 to 4×105 VG/cell. More than 1×1015 VG purified rAAVs can be easily obtained from 5 L suspension-cultured Sf9 cells. As expected, rAAV serotypes 1-13 show different potencies for in vitro transduction and celltype tropisms. Background: Recombinant adeno-associated virus (rAAV) has been widely used as an efficient transgenic vector in biomedical research, as well as gene therapy. Serotype-associated transduction efficiency, tissue- or cell-type tropism and immunological profile are major considerations in the various applications of rAAVs. There are increasing needs for different serotypes of rAAV, either naturally isolated or artificially engineered. However, affordable and scalable production of a desired serotype of rAAV remains very difficult, especially for researchers lacking relevant experience. Conclusion: In summary, the single BEV-derived OneBac system should prove popular for laboratory scaling-up production of any serotype of rAAV.

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