遗传学实验技术

Skin loss due to burns or ulcers is a major medical problem and is the motivation for the development of skin substitutes and skin replacement technologies, many of which have had some success in the clinic (1–4). Methods exist for the growth of large numbers of epidermal keratinocytes as well as dermal f ...

The ability of hematopoietic stem cells (HSCs) to engraft in a recipient and establish long-term repopulation of the hematopoietic system makes them ideal targets for gene therapy vectors designed to correct inherited or acquired diseases affecting the hematopoietic and immune sys ...

Antitumor immunity was first suggested in animals that reject tumor challenge after immunization with autologous inactivated tumor cells. Later, the discovery of tumor antigens recognized by T-cells strongly reinforced the concept that the tumor can be targeted by the immune system. ...

Most recent gene therapy protocols describe in vivo delivery of foreign genes by means of injecting adenoviral, adeno-associated, or lentiviral particles (1–4). Although these delivery systems show great promise, it is reasonable to believe that ex vivo gene therapy may move through cli ...

Nonviral gene delivery has great potential for replacement of recombinant protein therapy. In many cases, gene therapies would be a considerable improvement over existing therapies because of putative advantages in dosing schedule, patient compliance, toxicity, immunogeni ...

The development of gene transfer vectors from lentiviruses, such as the human immunodeficiency virus 1 (HIV-1), has opened exciting perspectives for the genetic treatment of a wide array of inherited and acquired diseases, because of their ability to achieve the efficient delivery, inte ...

Packaging systems based on human immunodeficiency virus type 1 (HIV-1) can be used to transfer genes (both ex vivo and in vivo in experimental animals), with high efficiency into a wide variety of cell types including nondividing and terminally differentiated cells such as muscle and neuronal ...

Lentiviruses, such as human immunodeficiency virus (HIV), feline immunodeficiency virus (FIV), and equine infectious anemia virus (EIAV), are members of the Retroviridae, viruses with enveloped capsids and a plus-stranded RNA genome. Like all retroviruses, the RNA genome of lentiv ...

The life cycle of retroviruses involves stable integration of viral genetic material into the host genome; expression of viral genes is, in part, regulated by host cell factors (1). These features make retroviruses a widely used efficient means for introducing foreign DNA into the cell genome. ...

Traditional onco-retroviral vector packaging systems, typified by those based on Moloney murine leukemia virus (MMLV), have many appealing features for gene transfer. MMLV vectors stably integrate their genome into their target cells, can package and carry up to 6.5 kb of foreign insert, c ...

Vector systems based on human immunodeficiency viruses (HIVs) (1) allow an efficient and stable gene transfer into nondividing cells (2,3). Because of the pathogenicity of the parental virus, the use of lentiviral vectors based on feline immunodeficiency virus (4,5), equine infectious ...

Sendai virus (SeV) is an enveloped virus with a nonsegmented negativestrand RNA genome of 15,384 nucleotides; it is a member of the Paramyxoviridae family (1–3). The virus is pneumotropic in rodent species such as mice and rats, but no pathogenicity has been reported in humans. SeV has been utilized f ...

Adenoviruses (Ads) are excellent mammalian gene transfer vectors because of their ability to infect efficiently a wide variety of quiescent and proliferating cell types from various species to direct high-level gene expression. Consequently, Ad vectors are extensively used as pot ...

Human adenovirus vectors have been used widely as gene delivery vehicles. However, one major problem related to the use of these vectors is the presence of a preexisting immunity to human adenoviruses in a majority of the population. We (1–3) and others (4–6) are therefore developing nonhuman ade ...

Adenoviruses (Ads) possess several features that make them attractive as mammalian gene transfer vectors. They can efficiently infect a wide variety of quiescent and proliferating cell types from various species to direct high-level viral gene expression, their 36-kb double-stra ...

The advantages of nonviral carriers are their ease of preparation and scaleup, capacity of DNA to be transferred, and safety in vivo. However, there also are disadvantages, including generally low efficiency and transience of transgene expression. To create more efficient systems, the u ...

Adeno-associated virus (AAV) is a nonpathogenic, replication-defective parvovirus that is being developed as a vector for human gene transfer. The recent interest in recombinant (r)AAV has been driven by the unexpected finding that these simple vectors can efficiently transduce a va ...

The adeno-associated virus (AAV) is a nonpathogenic member of the Parvoviridae family (for review,see ref. 1) Recently this virus has gained considerable interest and has been developed as a gene delivery vector (2). Six primate AAV serotypes (designated AAV types 1–6) have so far been identif ...

Gene transfer to the central nervous system (CNS) has shown major advances in recent years, with the development of novel vector systems and progress in basic virology (1–12). To improve gene transfer to CNS neurons, we have combined the critical elements of herpes simplex virus-1 (HSV-I) amplic ...

To circumvent the safety limitations of viral vectors and the cytotoxicity of liposomal carriers, several investigators have used receptor-targeted molecular conjugates to direct gene transfer into mammalian cells in vitro (1–26), and in vivo (27–40). This method has potential for hu ...