Packaging systems for human recombinant adenovirus to be used in

Chemistry: molecular biology and microbiology – Animal cell – per se ; composition thereof; process of...

Patent

Rate now

  [ 0.00 ] – not rated yet Voters 0   Comments 0

Details

435 691, 4353201, 435455, 424 9321, 536 231, C12N 1500

Patent

active

059941286

DESCRIPTION:

BRIEF SUMMARY
TECHNICAL FIELD

The invention relates to the field of recombinant DNA technology, more in particular to the field of gene therapy. In particular the invention relates to gene therapy using materials derived from adenovirus, in particular human recombinant adenovirus. It especially relates to novel virus derived vectors and novel packaging cell lines for vectors based on adenoviruses.


BACKGROUND

Gene therapy is a recently developed concept for which a wide range of applications can be and have been envisaged.
In gene therapy a molecule carrying genetic information is introduced into some or all cells of a host, as a result of which the genetic information is added to the host in a functional format.
The genetic information added may be a gene or a derivative of a gene, such as a cDNA, which encodes a protein. In this case the functional format means that protein can be expressed by the machinery of the host cell.
The genetic information can also be a sequence of nucleotides complementary to a sequence of nucleotides (be it DNA or RNA) present in the host cell. The functional format in this case is that the added DNA (nucleic acid) molecule or copies made thereof in situ are capable of base pairing with the complementary sequence present in the host cell.
Applications include the treatment of genetic disorders by supplementing a protein or other substance which is, through said genetic disorder, not present or at least present in insufficient amounts in the host, the treatment of tumors and (other) acquired diseases such as (auto)immune diseases or infections, etc.
As may be clear from the above, there are basically three different approaches in gene therapy, one directed towards compensating a deficiency present in a (mammalian) host; the second directed towards the removal or elimination of unwanted substances (organisms or cells) and the third towards application of a recombinant vaccine (tumors or foreign micro-organisms).
For the purpose of gene therapy, adenoviruses carrying deletions have been proposed as suitable vehicle. Adenoviruses are non-enveloped DNA viruses. Gene-transfer vectors derived from adenoviruses (so-called adenoviral vectors) have a number of features that make them particularly useful for gene transfer for such purposes. Eg. the biology of the adenoviruses is characterized in detail, the adenovirus is not associated with severe human pathology, the virus is extremely efficient in introducing its DNA into the host cell, the virus can infect a wide variety of cells and has a broad host-range, the virus can be produced in large quantities with relative ease, and the virus can be rendered replication defective by deletions in the early-region 1 (E1) of the viral genome.
The adenovirus genome is a linear double-stranded DNA molecule of approximately 36000 base pairs with the 55-kDa terminal protein covalently bound to the 5'terminus of each strand. The Ad DNA contains identical Inverted Terminal Repeats (ITR) of about 100 base pairs with the exact length depending on the serotype. The viral origins of replication are located within the ITRs exactly at the genome ends. DNA synthesis occurs in two stages. First, the replication proceeds by strand displacement, generating a daughter duplex molecule and a parental displaced strand. The displaced strand is single stranded and can form a so-called "panhandle" intermediate, which allows replication initiation and generation of a daughter duplex molecule. Alternatively, replication may proceed from both ends of the genome simultaneously, obviating the requirement to form the panhandle structure. The replication is summarized in FIG. 14 adapted from (Lechner and Kelly, 1977).
During the productive infection cycle, the viral genes are expressed in two phases: the early phase, which is the period upto viral DNA replication, and the late phase, which coincides with the initiation of viral DNA replication. During the early phase only the early gene products, encoded by regions E1, E2, E3 and E4, are expressed, which carry out a number of function

REFERENCES:
patent: 5378618 (1995-01-01), Sternberg et al.
patent: 5545522 (1996-08-01), Van Gelder et al.
patent: 5652224 (1997-07-01), Wilson et al.
patent: 5670488 (1997-09-01), Gregory et al.
patent: 5707618 (1998-01-01), Armentano et al.
patent: 5753500 (1998-05-01), Shenk et al.
Ngo et al., in The Protein Folding Problem and Tertiary Structure Prediction, 1994, Merz et al., (ed.), Birkhauser, Boston, MA, pp. 433 and 492-495, Jan. 27, 1997.
Engelhardt et al. (PNAS, vol. 91, pp. 6196-6200, 1994), Jan. 27, 1997.
Vanhaesebroeck et al. (Virology, (Jun. 1990) 176 (2) 362-8).
Armentano et al. (Human Gene Therapy 6:11343-1353, 1995).
Amalfitano et al, Proc. Natl. Acad. Sci. USA (Apr. 1996) 93: 3352-3356.
Amalfitano et al, Gene Therapy (1997) 4: 258-263.
Armentano et al, Human Gene Therapy (Oct. 1995) 6:1343-1353.
Brough et al, Journal of Virology (Sep. 1996) 70 (9):6497-6501.
Brough et al, Abstract Book CSH Conference On Gene Therapy (1996) 42.
Brough et al, Virology (1992) 190:624-634.
Caravokyri, et al, Journal of Virology (Nov. 1995) 69 (11):6627-6633.
Fallaux et al, Human Gene Therapy (1996) 7:215-222.
Fisher et al, Virology (1996) 217:11-22.
Gao et al, Journal of Virology (Dec. 1996) 70 (12):8934-8943.
Gorziglia et al, Journal of Virology (Jun. 1996) 70 (6):4173-4178.
Hardy et al, Journal of Virology (Mar. 1997) 71 (3):1842-1849.
Hehir et al, Journal of Virology (Dec. 1996) 70 (12):8459-8467.
Imler et al, Gene Therapy (1996) 3:75-84.
Krougliak et al, Human Gene Therapy (Dec. 1995) 6:1575-1586.
Lieber et al, Journal of Virology (Dec. 1996) 70:8944-8960.
Sabatie et al, Abstract Book 14th Meeting on Animal Cell Technology (1996) BI-3.
Schaack et al, Journal of Virology (Jul. 1995) 69 (7):4079-4085.
Wang et al, Gene Therapy (1995) 2:775-783.
Yeh et al, Journal of Virology (Jan. 1996) 70 (1):559-565.
Zhou, et al, Journal of Virology (Oct. 1996) 70 (1):7030-7038.

LandOfFree

Say what you really think

Search LandOfFree.com for the USA inventors and patents. Rate them and share your experience with other people.

Rating

Packaging systems for human recombinant adenovirus to be used in does not yet have a rating. At this time, there are no reviews or comments for this patent.

If you have personal experience with Packaging systems for human recombinant adenovirus to be used in, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Packaging systems for human recombinant adenovirus to be used in will most certainly appreciate the feedback.

Rate now

     

Profile ID: LFUS-PAI-O-1670599

  Search
All data on this website is collected from public sources. Our data reflects the most accurate information available at the time of publication.