Gene targeting in vivo by adeno‐associated virus vectors
Abstract
Therapeutic gene delivery typically involves the addition of a transgene expression cassette to mutant cells. This approach is complicated by transgene silencing, aberrant transcriptional regulation and insertional mutagenesis. An alternative strategy is to correct mutations through homologous recombination, allowing for normal regulation of gene expression from the endogenous locus. Adeno-associated virus (AAV) vectors containing single-stranded DNA efficiently transduce cells in vivo and have been shown to target homologous chromosomal sequences in cultured cells. To determine whether AAV-mediated gene targeting can occur in vivo, we developed a mouse model that contains a mutant, nuclear-localized lacZ gene inserted at the ubiquitously expressed ROSA26 locus. Foci of beta-galactosidase-positive hepatocytes were observed in these mice after injection with an AAV vector containing a lacZ gene fragment, and precise correction of the 4-bp deletion was demonstrated by gene sequencing. We also used AAV gene-targeting vectors to correct the naturally occurring GusB gene mutation responsible for murine mucopolysaccharidosis type VII.
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The initial gene repair frequency ranged from 1/6,300 to 1/11,600 hepatocytes and was within the expected range from previous experiments where selection with NTBC did not apply.
“After correction, the initial gene repair frequency ranged from 1/6,300 to 1/11,600 hepatocytes and was within the expected range from previous experiments15 where selection with NTBC did not apply.”
From Adeno‐associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo†‡ (2010) · cites this paper as [15]
The most notable handicap to gene repair therapy is the low frequency of correction in vivo.
“Most notable is the low frequency of correction in vivo.”
From Adeno‐associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo†‡ (2010) · cites this paper as [15]
To date, the frequency of gene repair correction is too low to be of therapeutic value for any disease.
“To date, this frequency is too low to be of therapeutic value for any disease.”
From Adeno‐associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo†‡ (2010) · cites this paper as [15]
In that study, a single point mutation in the β-glucuronidase gene was corrected at frequencies of 10−4 to 10−5 using AAV2 and AAV6 at 2 × 1011 to 6 × 1011 vg doses.
“In that study, a single point mutation in the β-glucuronidase gene was corrected at frequencies of 10−4 to 10−5 using AAV2 and AAV6 at 2 × 1011 to 6 × 1011 vg doses.”
From Adeno‐associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo†‡ (2010) · cites this paper as [15]
The low correction frequencies in that study were not therapeutic in treated mice because no selective advantage exists for corrected hepatocytes in that model.
“Nonetheless, the low correction frequencies were not therapeutic in treated mice, because no selective advantage exists for corrected hepatocytes in that model.”
From Adeno‐associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo†‡ (2010) · cites this paper as [15]
One report has shown limited efficacy in vivo using a neonatal mouse model of mucopolysaccharidosis type VII.
“One report has shown limited efficacy in vivo using a neonatal mouse model of the disease mucopolysaccharidosis type VII.15”
From Adeno‐associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo†‡ (2010) · cites this paper as [15]
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- Authors
- Miller, D. G., Wang, P.-R., Petek, L. M., Hirata, R. K., Sands, M. S., & Russell, D. W.
- Affiliations
- University of Washington (US)
- Washington University in St. Louis (US)
- Corresponding author
- Russell, David W
- Venue
- Nature Biotechnology
- Published
- 2006-07-30
- Volume
- 24
- Issue
- 8
- Pages
- 1022-1026
- ISSN
- 0733-222X (ISSN-L)
- 1087-0156
- 1546-1696
- OpenAlex
- W1993439119
- Cited by
- 105
- References
- 32
- Citations by year
- 2024: 2 · 2023: 1 · 2022: 2 · 2020: 6 · 2019: 5 · 2018: 5 · 2017: 5 · 2016: 9 · 2015: 9 · 2014: 6 · 2013: 3 · 2012: 12
- FWCI
- 4.73
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Nature Biotechnology (publishedVersion)
- PubMed (publishedVersion)
- Funders
- University of Washington
- National Institute of Diabetes and Digestive and Kidney Diseases (K08DK062100)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Keywords
- gene targeting, adeno-associated virus vectors, homologous recombination, in vivo gene therapy, lacZ, Rosa26 locus, beta-galactosidase, hepatocytes, mucopolysaccharidosis type VII, single-stranded DNA, transgene silencing, insertional mutagenesis, mouse model
- MeSH terms
- Adenoviridae, Animals, Genetic Engineering, Genetic Vectors, Mice, Inbred C57BL, Transfection, Gene Targeting, Mice
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