Table of Contents
TARGETED GENE THERAPY
(This post was generated from a computer program.
The contents of this post may change as the database is updated.)
Data topics will be presented in 3 primary formats.
Subject Matter
Principal Researcher
Time Period
With respect to Subject Matter -
the database is organized into the following Chapters -
(1) TGR-Non Disease Specific ( 44 records )
(2) Sickle Cell ( 10 records )
(3) Duchenne Muscular Dystrophy (DMD) ( 8 records )
(4) Crigler-Najjar Syndrome ( 6 records )
(5) Hepatic Genes ( 5 records )
(6) Plants ( 4 records )
(7) Hemophilia ( 4 records )
(8) CD - (Cardiovascular diseases) ( 3 records )
(9) TFO - (Triple helix-forming oligonucleotide) ( 2 records )
(10) Competition ( 2 records )
(11) Conference ( 2 records )
(12) EB - GABEB ( 2 records )
(13) Cancer ( 1 record )
(14) Miscellaneous ( 1 record )
(15) Regents ( 1 record )
(16) Retinal ( 1 record )
(17) Science ( 1 record )
(18) SFHR - (small fragment homologous replacement ) ( 1 record )
(19) SSO - (Single-stranded oligonucleotide) ( 1 record )
(20) Huntington's Disease ( 1 record )
Subject Matter Posts on Silicon Investor
SICKLE
Message 17993571
Duchenne Muscular Dystrophy (DMD)
Message 17993575
Crigler-Najjar Syndrome
Message 17993578
Hepatic Genes
Message 17993580
Hemophilia
Message 17993583
CD - (Cardiovascular diseases)
Message 17993588
Subject:TFO - (Triple helix-forming oligonucleotide)
Message 17993590
EB - GABEB
Message 17993591
SFHR - (small fragment homologous replacement )
Message 17993595
SSO - (Single-stranded oligonucleotide)
Message 17993597
TGR-Non Disease Specific (thru 2000)
Message 17993602
TGR-Non Disease Specific (after 2000)
Message 17993606
With respect to Principal Researchers -
the database contains the following principal researchers.
(1) Kmiec EB ( 24 records )
(2) Steer CJ ( 11 records )
(3) Yoon, Kyonggeun ( 6 records )
(4) Dickson, George J., Ph.D. ( 4 records )
(5) Kapsa R ( 3 records )
(6) Blaese, RM MD ( 2 records )
(7) Rando, Thomas M.D., Ph.D. ( 2 records )
(8) Bartlett RJ ( 2 records )
(9) Web Article ( 2 records )
(10) Baxter Health Care ( 2 records )
(11) Conferences ( 2 records )
(12) Lai LW ( 2 records )
(13) Goncz KK ( 2 records )
(14) Glazer PM ( 1 record )
(15) Gupta, S. ( 1 record )
(16) Jama ( 1 record )
(17) Kimeragen, Inc ( 1 record )
(18) Havre PA ( 1 record )
(19) Davidson College ( 1 record )
(20) Caldecott, Keith Dr ( 1 record )
(21) Brown, Brian PhD ( 1 record )
(22) Brown lain ( 1 record )
(23) Boatright,Jeffrey H., D ( 1 record )
(24) Blaese, Michael R. M.D. ( 1 record )
(25) Beetham PR ( 1 record )
(26) Aran JM. ( 1 record )
(27) Culver KW ( 1 record )
(28) Scheffer, H Dr ( 1 record )
(29) Wu XS ( 1 record )
(30) Welz C ( 1 record )
(31) Various DMD researchers ( 1 record )
(32) Valigen ( 1 record )
(33) Stevenson BJ ( 1 record )
(34) Steege, Gerrit van der ( 1 record )
(35) Stanojevic, D. ( 1 record )
(36) Sperber G ( 1 record )
(37) Lanzov VA ( 1 record )
(38) Science ( 1 record )
(39) Albuquerque-Silva J ( 1 record )
(40) Rugg, Elizabeth L Dr ( 1 record )
(41) Rothstein,Rodney ( 1 record )
(42) Porteous DJ ( 1 record )
(43) PETER R. BEETHAM ( 1 record )
(44) Olipitz W ( 1 record )
(45) Maurisse R ( 1 record )
(46) Lillicrap,David Dr. ( 1 record )
(47) Li,Xianfeng ( 1 record )
(48) Li ZH ( 1 record )
(49) Sharrer, Terry G. ( 1 record )
BUZZ_WORDS.....(subject to errors of understanding)
Chimeraplast
The prototypic structure of the chimeraplast was a contiguous stretch of 68 nucleotides containing both RNA and DNA residues. One strand of the heteroduplex structure consisted of a central pentameric block of DNA bases flanked on either side by ten 29-O-methylated RNA residues providing increased resistance to RNaseH enzymatic activity. The complementary strand consisted of all DNA bases. Polythymidine hairpins were placed at both ends to provide stability for secondary structure and a 39 GC clamp to increase nuclease resistance. The 39 and 59 ends were juxtaposed but not joined to allow unwinding of the chimeraplast and greater flexibility to interact with its target sequence and repair proteins.
Chimeric
reflect the fact the molecule consists of different kinds of genetic material
Chimeric RNA/DNA oligonucleotide
See Chimeraplast
Chimeric oligonucleotides
See Chimeraplast
Targeted Gene Repair
oligos used to force repair mechanisms to change DNA sequences
RDO
RNA/DNA oligonucleotides
Chimeraplasty
Targeted gene correction
Oligo
synthetic paperclip-shaped oligonucleotide
Sickle cell anemia
a single letter misspelled, in the gene encoding the b-globin strand of hemoglobin
Cystic fibrosis
Over 70 percent of the cases of cystic fibrosis are attributable to the deletion of three nucleotides in the CFTR gene
Muscular dystrophy
Lack of a protein called dystrophin
Crigler-Najjar syndrome
crucial enzyme UGT is either missing or is decreased in quantity or activity
Generalised Atrophic Benign Epidermolysis Bullosa (GABEB)
genetic disorders causing blistering and shearing of the skin from even the gentlest friction, often from everyday activities
Gene
Length of DNA that codes for a protein
Nucleotides
adenine (A),
Nucleotides
thymine (T),
Nucleotides
cytosine (C),
Nucleotides
guanine (G)
Nucleotides
and uracil (U).
DNA
ATCG
RNA
ACGU
Adenoviruses
no integration / transient
Adeno-associated virus (AAV)
are non-pathogenic human parvoviruses, dependant on a helper virus, usually adenovirus, to proliferate. They are capable of infecting both dividing & non dividing cells, & in the absence of a helper virus integrate into a specific point of the host genome (19q 13-qter) at a high frequency (Kotin et al, 1990).
Retroviruses
disarmed or "gutted" / no control over
Lipofection - liposomes
tiny membrane-bound spheres loaded with DNA
Gene Targeting
replacement of native (bad) copy of gene with a corrected copy
Targeted Gene Repair
oligos used to force repair mechanisms to change DNA sequences
Antisense and RNAi technology
can inhibit expression of genes very selectively
Overexpress mutant gene
insert somewhere and crank out good protein
Electroporation
Zap them just like E. coli
John McCarthy |
