Results 21 to 30 of about 2,093,614 (359)

Progress in the use of adeno-associated viral vectors for gene therapy [PDF]

, 2004
The development of safe and efficient gene transfer vectors is crucial for the success of gene therapy trials. A viral vector system promising to meet these requirements is based on the apathogenic adeno-associated virus (AAV-2), a member of the ...
Braun-Falco, M., Buning, H., Hallek, M.
core   +1 more source

Data collection infrastructure for patient outcomes in the UK – opportunities and challenges for cell and gene therapies launching

Journal of Market Access & Health Policy, 2019
Background: Cell and gene therapies are associated with uncertainty around their value claims at launch due to limitations of supporting clinical data; furthermore, their high costs present affordability issues for payers.
Jesper Jørgensen, Laura Mungapen, Panos Kefalas   +2 more
doaj   +1 more source

Impaired regeneration in calpain-3 null muscle is associated with perturbations in mTORC1 signaling and defective mitochondrial biogenesis

Skeletal Muscle, 2017
Background Previous studies in patients with limb-girdle muscular dystrophy type 2A (LGMD2A) have suggested that calpain-3 (CAPN3) mutations result in aberrant regeneration in muscle.
Mehmet E. Yalvac, Jakkrit Amornvit, Cilwyn Braganza, Lei Chen, Syed-Rehan A. Hussain, Kimberly M. Shontz, Chrystal L. Montgomery, Kevin M. Flanigan, Sarah Lewis, Zarife Sahenk   +9 more
doaj   +1 more source

Circumventing antivector immunity: potential use of nonhuman adenoviral vectors [PDF]

, 2014
Adenoviruses are efficient gene delivery vectors based on their ability to transduce a wide variety of cell types and drive high-level transient transgene expression. While there have been advances in modifying human adenoviral (HAdV) vectors to increase
Beard C.W., Benkö M., Cauthen A.N., Crawford-Miksza L., Estrella Lopez-Gordo, Gall J., Gall J.G., Gattoni A., Ginsberg H.S., Haviv Y.S., Hofmann C., Iva I. Podgorski, Kajon A.E., Kanerva A., Kass-Eisler A., Kuriyama S., Mastrangeli A., Michou A.I., Nicholas Downes, Ramon Alemany, Rogozhin V.N., Roy S., Seiradake E., Shmarov M.M., Soudais C., Tutykhina I.L., Wang D.Y., Wohlfart C., Ying B., Zakhartchouk A.N., Zeng Q.   +30 more
core   +3 more sources

Human Cytomegalovirus Induces Cellular and Humoral Virus-specific Immune Responses in Humanized BLT Mice

Scientific Reports, 2017
The strict species specificity of Human Cytomegalovirus (HCMV) has impeded our understanding of antiviral adaptive immune responses in the context of a human immune system.
Lindsey B. Crawford, Rebecca Tempel, Daniel N. Streblow, Craig Kreklywich, Patricia Smith, Louis J. Picker, Jay A. Nelson, Patrizia Caposio   +7 more
doaj   +1 more source

Functional roles of the membrane-associated AAV protein MAAP

Scientific Reports, 2021
With a limited coding capacity of 4.7 kb, adeno-associated virus (AAV) genome has evolved over-lapping genes to maximise the usage of its genome. An example is the recently found ORF in the cap gene, encoding membrane-associated accessory protein (MAAP),
Lionel Galibert, Amira Hyvönen, Reetta A. E. Eriksson, Salla Mattola, Vesa Aho, Sami Salminen, Justin D. Albers, Sanna K. Peltola, Saija Weman, Tiina Nieminen, Seppo Ylä-Herttuala, Hanna P. Lesch, Maija Vihinen-Ranta, Kari J. Airenne   +13 more
doaj   +1 more source

Incorporation of aptamers in the terminal loop of shRNAs yields an effective and novel combinatorial targeting strategy. [PDF]

, 2017
Gene therapy by engineering patient's own blood cells to confer HIV resistance can potentially lead to a functional cure for AIDS. Toward this goal, we have previously developed an anti-HIV lentivirus vector that deploys a combination of shRNA, ribozyme ...
Castanotto, Daniela, Li, Haitang, Pang, Ka Ming, Rossi, John J, Scherer, Lisa   +4 more
core   +1 more source

Stochastic models and numerical algorithms for a class of regulatory gene networks [PDF]

, 2008
Regulatory gene networks contain generic modules like those involving feedback loops, which are essential for the regulation of many biological functions.
Fournier, Thomas, Gabriel, Jean-Pierre, Galbete, Jose, Mazza, Christian, Mermod, Nicolas, Pasquier, Jerome   +5 more
core   +2 more sources

Gene therapy

Biology of Blood and Marrow Transplantation, 1999
For the past two decades, concerted efforts have been made to treat human disease by replacing nonfunctioning genes in cells or by correcting mutations that produce disease. Successful application of these methods could lead to effective therapies for a variety of genetic and acquired diseases, many of which are not treatable today.
openaire   +2 more sources

Cochlear Gene Therapy [PDF]

Cold Spring Harbor Perspectives in Medicine, 2012
Over 450 million people worldwide suffer from hearing loss, leading to an estimated economic burden of ∼$750 billion. The past decade has seen significant advances in the understanding of the molecular mechanisms that contribute to hearing, and the environmental and genetic factors that can go awry and lead to hearing loss.
Lawrence R, Lustig, Omar, Akil
openaire   +4 more sources