Gene therapy: adenovirus vectors

doi:10.1016/0959-437X(93)90126-A

Current Opinion in Genetics & Development

Volume 3, Issue 3, June 1993, Pages 499-503

https://doi.org/10.1016/0959-437X(93)90126-A Get rights and content

Abstract

The past year has seen a proliferation in the use of recombinant, replication-defective adenoviruses for experimental models of gene therapy. The fact that adenovirus infects most cell types with no requirement for cell division, combined with the high titers and high efficiency of gene transfer obtainable with recombinant adenovirus, make it a promising system for in vivo human gene therapy.

References (31)

J.M. Wilson et al.
Hepatocyte-Directed Gene Transfer In Vivo Leads to Transient Improvement of Hypercholesterolemia in Low Density Lipoprotein Receptor-Deficient Rabbits
J Biol Chem
(1992)
J.M. Wilson et al.
A Novel Mechanism for Achieving Transgene Persistence In Vivo after Somatic Gene Transfer into Hepatocytes
J Biol Chem
(1992)
M.A. Rosenfeld et al.
In Vivo Transfer of the Human Cystic Fibrosis Transmembrane Conductance Regulator Gene to the Airway Epithelium
Cell
(1992)
A.D. Miller
Human Gene Therapy Comes of Age
Nature
(1992)
J.M. Wilson et al.
Temporary Amelioration of Hyperlipidemia in Low Density Lipoprotein Receptor-Deficient Rabbits Transplanted with Genetically Modified Hepatocytes
K. Yoshimura et al.
Expression of the Human Cystic Fibrosis Transmembrane Conductance Regulator Gene in the Mouse Lung After In Vivo Intratracheal Plasmid-Mediated Gene Transfer
Nucleic Acids Res
(1992)
E.G. Nabel et al.
Site-Specific Gene Expression In Vivo by Direct Gene Transfer into the Arterial Wall
Science
(1990)
C.S. Lim et al.
Direct In Vivo Gene Transfer into the Coronary and Peripheral Vasculatures of the Intact Dog
Circulation
(1991)
J.A. Wolff et al.
Direct Gene Transfer into Mouse Muscle In Vivo
Science
(1991)
G. Acsadi et al.
Direct Gene Transfer and Expression into Rat Heart In Vivo
New Biol
(1991)

G. Acsadi et al.

Human Dystrophin Expression in mdx Mice After Intramuscular Injection of DNA Constructs

Nature

(1991)

S.E. Strauss

Adenovirus Infection in Humans

R.M. Chanock et al.

Immunization by Selective Infection with Type 4 Adenovirus Grown in Human Diploid Tissue Culture. I. Safety and Lack of Oncogenicity and Tests for Potency in Volunteers

J Am Med Assoc

(1966)

K.L. Berkner

Development of Adenovirus Vectors for the Expression of Heterologous Genes

Biotechniques

(1988)

M.A. Rosenfeld et al.

Adenovirus-Mediated Transfer of a Recombinant α1-Antitrypsin Gene to the Lung Epithelium In Vivo

Science

(1991)

Cited by (402)

The gap between development and manufacturing in gene therapy: Strategic options for overcoming traps
2023, Drug Discovery Today
Gene therapy has been one of the most promising therapeutic approaches in recent years. This study analyzed a research and development (R&D) system for adeno-associated virus (AAV)-based gene therapies, and confirmed that there was a gap between the development and manufacturing capabilities. Although a start-up company that has no academic or manufacturing facilities can begin the clinical development process, it cannot successfully continue development activities without forming alliances and capital investment or, at a certain stage, without appropriate manufacturing and marketing strategies. We reviewed a series of case studies to categorize the acquisition patterns of pharmaceutical companies that are engaged in AAV gene therapy. These results provide insights into the R&D structures for AAV gene therapies from a technological management perspective.
Development of a recombinant adenovirus-vectored vaccine against both infectious hematopoietic necrosis virus and infectious pancreatic necrosis virus in rainbow trout (Oncorhynchus mykiss)
2023, Fish and Shellfish Immunology
Citation Excerpt :
Replication-defective recombinant adenoviruses are extensively used as vectored vaccines for mammalian pathogens [31–36]. The best advantage of replication-defective recombinant adenovirus vectors is that they can efficiently transfer target genes into host cells by immersion vaccination inducing potent and sustained specific humoral and cellular immune responses, but the transferred target genes are not required to be integrated into host genome [32,37]. Interestingly, several kinds of teleost cell lines, including rainbow trout cells can be infected by a recombinant adenovirus [38].
Infectious hematopoietic necrosis virus (IHNV) and infectious pancreatic necrosis virus (IPNV) are typical pathogens of rainbow trout Oncorhynchus mykiss, and the concurrent infection of the two viruses is very common among modern trout hatcheries, which has caused huge economic losses to the rainbow trout farming industry. To prevent and control the spread of IHNV and IPNV in juvenile trout simultaneously, in this study a bivalent recombinant adenovirus vaccine with IHNV Glycoprotein (G) and IPNV VP2 genes was developed. After immunizing juvenile trout with this bivalent vaccine via the immersion route, the expression levels of IHNV G and IPNV VP2 and the representative immune genes in vaccinated and control rainbow trout were tested to evaluate the correlation of immune responses with the expression of viral genes. The neutralizing antibody level induced by this bivalent vaccine as well as the protection efficacy of the vaccine against IHNV and IPNV was also evaluated. The results showed that IHNV G and IPNV VP2 were successfully expressed in juvenile trout, and all the innate and adaptive immune genes were up-regulated. This indicated that the level of the innate and adaptive immune responses were significantly increased, which might be induced by the high expression of the two viral proteins. Compared with the controls, high levels of neutralizing antibodies against IHNV and IPNV were induced in the vaccinated trout. Besides, the bivalent recombinant adenovirus vaccine showed high protection rate against IHNV, with the relative percent survival (RPS) of 81.25%, as well as against IPNV, with the RPS of 78.95%. Taken together, our findings clearly demonstrated that replication-defective adenovirus can be developed as a qualified vector for fish vaccines and IHNV G and IPNV VP2 were two suitable antigenic genes that could induce effective immune protection against these two pathogens. This study provided new insights into developing bivalent vectored vaccines and controlling the spread of IHNV and IPNV simultaneously in juvenile trout.
Material-based engineering of bacteria for cancer diagnosis and therapy
2021, Applied Materials Today
Various categories of biomaterials have been utilized for drug delivery, genetic modification, photodynamic and photothermal therapies due to their distinct physicochemical properties, including photothermal convertibility, stimuli-responsiveness, and inherent capability to generate photodynamical radicals. However, successful treatments of cancer are largely hindered by the limited accessibility of nanomaterials into hypoxic or metastatic tumor tissues. Among the various tumor-targeting strategies, bacterial fabrication exhibits particular advantages such as specific hypoxia tropism, high motility, and rapid self-replication. Biomineralization, i.e. bacterial modification, involves the fabrication of bacteria by nanomaterials for precise cancer imaging as well as targeted drug delivery, overcoming the physiological barriers and improving the therapeutic efficiency. Fabrication of bacteria strains can be conducted by various methods, including direct adsorption, electrostatic interaction, covalent ligation, and surface precipitation. In this review, a brief introduction to commonly-utilized biomaterials and bacteria species is provided. A systemic overview of recent advances of bacteria fabrication strategies and techniques are then discussed, followed by future prospective of bacteria-facilitated cancer therapy and diagnostics.
CRISPR-Cas9 in cancer therapeutics
2021, Progress in Molecular Biology and Translational Science
Cancer is a disease mainly caused by an accumulation of mutations in cells. Consequently, correcting those genetic aberrations could be a potential treatment strategy. The traditional route for cancer drug development is tedious, laborious, and time-consuming. Due to target identification, drug formulation, pre-clinical testing, clinical testing, and regulatory hurdles, on average, it takes 10–15 years for a cancer drug to go from target discovery to a marketable oncology drug. The advent of CRISPR-Cas9 technology has greatly expedited this procedure. CRISPR-Cas9 has single-handedly accelerated target identification and pre-clinical testing. Furthermore, CRISPR-Cas9 has also been used in ex vivo editing of T-cells to specifically target tumor cells. In this chapter, we will discuss the various ways in which CRISPR-Cas9 has been used for the betterment of the cancer drug development process. Additionally, we will discuss various ways in which it is currently being used as therapy and the drawbacks which restrict the use of this groundbreaking technology as direct therapy.
The evolution and history of gene editing technologies
2021, Progress in Molecular Biology and Translational Science
Scientific enquiry must be the driving force of research. This sentiment is manifested as the profound impact gene editing technologies are having in our current world. There exist three main gene editing technologies today: Zinc Finger Nucleases, TALENs and the CRISPR-Cas system. When these systems were being uncovered, none of the scientists set out to design tools to engineer genomes. They were simply trying to understand the mechanisms existing in nature. If it was not for this simple sense of wonder, we probably would not have these breakthrough technologies. In this chapter, we will discuss the history, applications and ethical issues surrounding these technologies, focusing on the now predominant CRISPR-Cas technology. Gene editing technologies, as we know them now, are poised to have an overwhelming impact on our world. However, it is impossible to predict the route they will take in the future or to comprehend the full impact of its repercussions.
Development of a live vector vaccine against infectious pancreatic necrosis virus in rainbow trout
2020, Aquaculture
Infectious pancreatic necrosis virus (IPNV) causes severe viral disease in salmonids, with major economic losses in the global rainbow trout aquaculture industry following from a high mortality rate. In the present study, we aimed to develop a safe and efficient vaccine to protect rainbow trout against IPNV infection. Administered via the immersion route, a live vector vaccine containing the coding region of the IPNV VP2 induced protective immune responses in rainbow trout. For best protection, juvenile rainbow trout should be immunized with a 1:100 dilution of the titer with 1 × 10^10.0 ml⁻¹ TCID₅₀ of the live vector vaccine, via 10-min immersion. Expression of the IPNV VP2 gene was confirmed in spleens of vaccinated rainbow trout, and reached the highest level at 3 days post-vaccination (dpv) and thereafter gradually decreased between 3 and 15 dpv. The expression of TLR-3, TLR-7 and TLR-8 was upregulated after vaccination, and the highest expression levels of IFN-1, Mx-1, Mx-3, Vig-1 and Vig-2 were detected at 3 dpv. Four markers of the adaptive immune response (CD4, CD8, IgM and IgT) were continuously increased in the spleens of vaccinated fish as compared with controls, between 3 and 15 dpv. The cumulative percentage mortality differed significantly between the vaccinated fish and the controls (empty-plasmid-vaccinated). The results of the IPNV challenge showed that the live vector vaccine had a high protection rate against IPNV (relative percent survival value of 88.24), and the vaccinated rainbow trout displayed high levels of serum antibodies against IPNV infection. Taken together, our results demonstrate that this is a promising live vector vaccine that could be used to protect rainbow trout against IPNV infection.

View all citing articles on Scopus

View full text

Gene therapy: adenovirus vectors

Abstract

J Biol Chem

J Biol Chem

Cell

Human Gene Therapy Comes of Age

Nature

Temporary Amelioration of Hyperlipidemia in Low Density Lipoprotein Receptor-Deficient Rabbits Transplanted with Genetically Modified Hepatocytes

Expression of the Human Cystic Fibrosis Transmembrane Conductance Regulator Gene in the Mouse Lung After In Vivo Intratracheal Plasmid-Mediated Gene Transfer

Nucleic Acids Res

Site-Specific Gene Expression In Vivo by Direct Gene Transfer into the Arterial Wall

Science

Direct In Vivo Gene Transfer into the Coronary and Peripheral Vasculatures of the Intact Dog

Circulation

Direct Gene Transfer into Mouse Muscle In Vivo

Science

Direct Gene Transfer and Expression into Rat Heart In Vivo

New Biol

Human Dystrophin Expression in mdx Mice After Intramuscular Injection of DNA Constructs

Nature

Adenovirus Infection in Humans

Immunization by Selective Infection with Type 4 Adenovirus Grown in Human Diploid Tissue Culture. I. Safety and Lack of Oncogenicity and Tests for Potency in Volunteers

J Am Med Assoc

Development of Adenovirus Vectors for the Expression of Heterologous Genes

Biotechniques

Adenovirus-Mediated Transfer of a Recombinant α1-Antitrypsin Gene to the Lung Epithelium In Vivo

Science