Gene Transfer Techniques

Publication Title: 
Methods in Molecular Biology (Clifton, N.J.)

Intrauterine gene therapy (IUGT) potentially enables the treatment and possible cure of monogenic -diseases that cause severe fetal damage. The main benefits of this approach will be the ability to correct the disorder before the onset of irreversible pathology and inducing central immune tolerance to the vector and transgene if treatment is instituted in early gestation.

Author(s): 
Mattar, Citra N.
Biswas, Arijit
Choolani, Mahesh
Chan, Jerry K. Y.
Publication Title: 
Molecular Therapy: The Journal of the American Society of Gene Therapy

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by the progressive loss of motor neurons in the brain and spinal cord. We have recently shown that human mesenchymal stem cells (hMSCs) modified to release glial cell line-derived neurotrophic factor (GDNF) decrease disease progression in a rat model of ALS when delivered to skeletal muscle. In the current study, we determined whether or not this effect could be enhanced by delivering GDNF in concert with other trophic factors.

Author(s): 
Krakora, Dan
Mulcrone, Patrick
Meyer, Michael
Lewis, Christina
Bernau, Ksenija
Gowing, Genevieve
Zimprich, Chad
Aebischer, Patrick
Svendsen, Clive N.
Suzuki, Masatoshi
Publication Title: 
Nucleic Acids Research

The rRNA genes in the somatic macronucleus of Tetrahymena thermophila are normally on 21 kb linear palindromic molecules (rDNA). We examined the effect on rRNA gene dosage of transforming T.thermophila macronuclei with plasmid constructs containing a pair of tandemly repeated rDNA replication origin regions unlinked to the rRNA gene. A significant proportion of the plasmid sequences were maintained as high copy circular molecules, eventually consisting solely of tandem arrays of origin regions.

Author(s): 
Pan, W. J.
Blackburn, E. H.
Publication Title: 
Nucleic Acids Research

The rRNA genes in the somatic macronucleus of Tetrahymena thermophila are normally on 21 kb linear palindromic molecules (rDNA). We examined the effect on rRNA gene dosage of transforming T.thermophila macronuclei with plasmid constructs containing a pair of tandemly repeated rDNA replication origin regions unlinked to the rRNA gene. A significant proportion of the plasmid sequences were maintained as high copy circular molecules, eventually consisting solely of tandem arrays of origin regions.

Author(s): 
Pan, W. J.
Blackburn, E. H.
Publication Title: 
Biological Psychiatry

BACKGROUND: Marijuana use by teenagers often predates the use of harder drugs, but the neurobiological underpinnings of such vulnerability are unknown. Animal studies suggest enhanced heroin self-administration (SA) and dysregulation of the endogenous opioid system in the nucleus accumbens shell (NAcsh) of adults following adolescent ?(9)-tetrahydrocannabinol (THC) exposure. However, a causal link between proenkephalin (Penk) expression and vulnerability to heroin has yet to be established.

Author(s): 
Tomasiewicz, Hilarie C.
Jacobs, Michelle M.
Wilkinson, Matthew B.
Wilson, Steven P.
Nestler, Eric J.
Hurd, Yasmin L.
Publication Title: 
BMC medical ethics

BACKGROUND: Ocular gene transfer clinical trials are raising hopes for blindness treatments and attracting media attention. News media provide an accessible health information source for patients and the public, but are often criticized for overemphasizing benefits and underplaying risks of novel biomedical interventions. Overly optimistic portrayals of unproven interventions may influence public and patient expectations; the latter may cause patients to downplay risks and over-emphasize benefits, with implications for informed consent for clinical trials.

Author(s): 
Benjaminy, Shelly
Bubela, Tania
Publication Title: 
Cardiovascular & Hematological Disorders Drug Targets

Previous studies using different techniques have shown that adenoviral-mediated gene transfer to different tissues, including the kidney, is more efficient in neonatal mice. In this study, we report a simple technique that allows an efficient and long term expression of beta-galactosidase (beta-gal) in the heart of newborn mice. Newborn and adult C57BL6/J mice were subjected to a single retro-orbital venous plexus injection of recombinant adenoviral vectors (rAd) (2 x 10(9) particles/g body weight) carrying the lac Z gene.

Author(s): 
Jerebtsova, Marina
Ye, Xuehai
Ray, Patricio E.
Publication Title: 
PloS One

Genetically modified hematopoietic progenitors represent an important testing platform for a variety of cell-based therapies, pharmaceuticals, diagnostics and other applications. Stable expression of a transfected gene of interest in the cells is often obstructed by its silencing. DNA transposons offer an attractive non-viral alternative of transgene integration into the host genome, but their broad applicability to leukocytes and other "transgene unfriendly" cells has not been fully demonstrated.

Author(s): 
Mossine, Valeri V.
Waters, James K.
Hannink, Mark
Mawhinney, Thomas P.
Publication Title: 
Molecular Therapy: The Journal of the American Society of Gene Therapy

Many metabolic diseases are caused by defects in the metabolic pathways in the liver. Others result from the absence of specific proteins normally produced and secreted by the liver. Because these metabolic disorders are usually caused by single gene defect, they are ideal candidates for gene therapy. We have previously shown that mouse liver can be transfected by mechanically massaging the liver (MML) after intravenous injection of naked plasmid DNA.

Author(s): 
Liu, Feng
Lei, Jing
Vollmer, Regis
Huang, Leaf
Publication Title: 
FEBS letters

The potential for transfer of antibiotic resistance genes from genetically modified (GM) plant material to microbes through genetic recombination in the human or animal gut is a consideration that has engendered caution in the use of GM foods. This study was aimed at defining the optimal physical and chemical conditions necessary to ensure sufficient fragmentation of DNA in plant tissues to a size where it would be unlikely to be stably transferred to bacterial gut microflora.

Author(s): 
Chiter, A.
Forbes, J. M.
Blair, G. E.

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