Results 31 to 40 of about 1,824,389 (266)

Gene Therapy

Hematology/Oncology Clinics of North America, 2017
Gene therapy refers to a rapidly growing field of medicine in which genes are introduced into the body to treat or prevent diseases. Although a variety of methods can be used to deliver the genetic materials into the target cells and tissues, modified viral vectors represent one of the more common delivery routes because of its transduction efficiency ...
Daniel E, Bauer, Donald B, Kohn
openaire   +4 more sources

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

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

Amplification of the androgen receptor may not explain the development of androgen-independent prostate cancer [PDF]

, 2001
Objective To examine the role of androgen receptor (AR) gene amplification and aneusomy of the X chromosome in the development of antiandrogen-resistant prostate cancer.
Bartlett, J.M.S., Edwards, J., Krishna, N.S., Mukherjee, R., Underwood, M.A., Watters, A.D.   +5 more
core   +1 more source

Alphaviruses in Gene Therapy [PDF]

Viruses, 2009
Alphaviruses are enveloped single stranded RNA viruses, which as gene therapy vectors provide high-level transient gene expression. Semliki Forest virus (SFV), Sindbis virus (SIN) and Venezuelan Equine Encephalitis (VEE) virus have been engineered as efficient replication-deficient and -competent expression vectors.
openaire   +7 more sources

Non-viral approaches to gene therapy [PDF]

, 1993
Several advances in non-viral gene transfer technology have been reported over the past year. Cationic lipids have been successfully used to deliver genes in vivo, providing a clear alternative to recombinant viruses.
Behr, Behr, Blumenthal, Böttger, Cotten, Cotten, Cotten, Cristiano, Curiel, Demeneix, Ellens, Felgner, Felgner, Fitzgerald, Haensler, Haensler, Hawley-Nelson, Huckett, Hyde, Kaneda, Legendre, Leonetri, Midoux, Nabel, Parente, Plank, Plautz, Rosenkranz, Seth, Shen, Stribling, Subbarao, Tang, Tomita, Trubetskoy, Trubetskoy, Ulmer, Wagner, Wagner, Wagner, Wagner, Wagner, Williams, Wu, Zhu   +44 more
core   +1 more source

Graph Frequency Features of Cancer Gene Co-Expression Networks [PDF]

arXiv, 2023
Complex gene interactions play a significant role in cancer progression, driving cellular behaviors that contribute to tumor growth, invasion, and metastasis. Gene co-expression networks model the functional connectivity between genes under various biological conditions.
arxiv  

Identification and validation of targets for cancer immunotherapy: from the bench-to-bedside [PDF]

, 2013
Chapter 12 in Novel Gene Therapy ...
Brooks, Suzanne E., Denniss, Frances, Guinn, Barbara-Ann, Khan, Ghazala, Sigurdardottir, Dagmar   +4 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