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Gene Therapy

journal
https://www.readbyqxmd.com/read/28737744/delivering-advanced-therapies-the-big-pharma-approach
#1
REVIEW
J Tarnowski, D Krishna, L Jespers, A Ketkar, R Haddock, J Imrie, S Kili
After two decades of focused development and some recent clinical successes, cell and gene therapy (CGT) is emerging as a promising approach to personalized medicines. Genetically engineered cells as a medical modality are poised to stand alongside or in combination with small molecule and biopharmaceutical approaches to bring new therapies to patients globally. Big pharma can play a vital role in industrializing CGT by focusing on diseases with high un-met medical need and compelling genetic evidence. Pharma should invest in manufacturing and supply chain solutions that deliver reproducible, high quality therapies at a commercially viable cost...
July 24, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28737743/development-of-gene-therapies-lessons-from-nusinersen
#2
REVIEW
L Xu, I Irony, W W Bryan, B Dunn
The nusinersen development and approval process provide important lessons regarding the pathway to marketing approval for gene therapies. These lessons emphasize rigorous clinical trial design, flexibility in trial design and analysis, a collaborative effort with regular communications between the drug developer and the FDA, and use of FDA's expedited programs. These lessons are critical to the development of gene therapies for the treatment of serious or life-threatening rare diseases.Gene Therapy accepted article preview online, 24 July 2017...
July 24, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28726798/gene-therapy-research-in-asia
#3
REVIEW
Hong-Xin Deng, Yuan Wang, Qiu-Rong Ding, Da-Li Li, Yu-Quan Wei
Gene therapy has shown great potential for the treatment of diseases that previously were either untreatable or treatable but not curable with conventional schemes. Recent progress in clinical gene therapy trials has emerged in various severe diseases, including primary immunodeficiencies, leukodystrophies, Leber's congenital amaurosis, haemophilia as well as retinal dystrophy. The clinical transformation and industrialization of gene therapy in Asia have been remarkable and continue making steady progress...
July 20, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28692018/the-state-of-gene-therapy-research-in-africa-its-significance-and-implications-for-the-future
#4
REVIEW
P Arbuthnot, M B Maepa, A Ely, M S Pepper
Gene therapy has made impressive recent progress and has potential for treating a wide range of diseases, many of which are important to Africa. However, as a result of lack of direct public funding and skilled personnel, direct research on gene therapy in Africa is currently limited and resources to support the endeavor are modest. A strength of the technology is that it is based on principles of rational design, and the tools of gene therapy are now highly versatile. For example gene silencing and gene editing may be used to disable viral genes for therapeutic purposes...
July 10, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28682314/nasal-application-of-hsv-encoding-human-preproenkephalin-blocks-craniofacial-pain-in-a-rat-model-of-traumatic-brain-injury
#5
A C Meidahl, M Klukinov, A Z Tzabazis, J C Sorensen, D C Yeomans
According to Centers for Disease Control and Prevention, each year, an estimated 1.7 million Americans sustain a traumatic brain injury (TBI), which frequently leads to chronic craniofacial pain. In this study we examine a gene therapy approach to the treatment of post-TBI craniofacial neuropathic pain using nasal application of a Herpes Simplex Virus-based vector expressing human proenkephalin (SHPE) to target the trigeminal ganglia. Mild TBI was induced in rats by use of a modified Fluid Percussion Model...
July 6, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28681841/recombinant-elastin-based-nanoparticles-for-targeted-gene-therapy
#6
D A Monfort, P Koria
Among viruses, lentiviral vectors have been popular vectors for gene delivery due to their efficient mode of gene delivery. However, the non-specific delivery of genes associated with lentiviral vectors may result in undesirable side effects. Here, we propose a heterogeneous nanoparticle delivery system for targeted delivery of lentiviral particles containing a therapeutic gene. The heterogeneous nanoparticles consist of the low density lipoprotein receptor 3 (LDLR3) and the keratinocyte growth factor (KGF), each fused to elastin-like-polypeptides (ELPs), LDLR3-ELP and KGF-ELP, respectively...
July 5, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28660888/use-of-a-gamma-2-herpesvirus-as-a-vector-to-deliver-antibodies-to-rhesus-monkeys
#7
G F Bischof, Y C Shin, S P Fuchs, J M Martinez-Navio, W A Lauer, E G Rakasz, R C Desrosiers
The gamma-2 herpesvirus of rhesus monkeys, rhesus monkey rhadinovirus (RRV), persists principally in B cells of its host. We constructed recombinant strains of RRV expressing the rhesus monkey-derived anti-SIV monoclonal antibodies 4L6 and 5L7 and compared the RRV-mediated in vivo delivery of these antibodies in rhesus monkeys with previous studies that utilized intramuscular delivery with an adeno-associated virus (AAV) vector. Recombinant RRV-4L6 and RRV-5L7 were both shown to stably produce the antibodies in persistently infected B-cell lines in culture...
June 29, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28644430/the-clinical-landscape-for-sma-in-a-new-therapeutic-era
#8
REVIEW
K Talbot, E F Tizzano
Despite significant advances in basic research, the treatment of degenerative diseases of the nervous system remains one of the greatest challenges for translational medicine. The childhood onset motor neuron disorder spinal muscular atrophy (SMA) has been viewed as one of the more tractable targets for molecular therapy, due to a detailed understanding of the molecular genetic basis of the disease. In SMA, inactivating mutations in the SMN1 gene can be partially compensated for by limited expression of SMN protein from a variable number of copies of the SMN2 gene, which provides both a molecular explanation for phenotypic severity and a target for therapy...
June 23, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28639617/gene-therapy-for-spinomuscular-atrophy-a-biomedical-advance-a-missed-opportunity-for-more-equitable-drug-pricing
#9
T Friedmann
An experimental approach for gene therapy of spinomuscular atrophy has been reported to prevent development of the neuromuscular features of this lethal and previously untreatable disorder. The approach involves treatment of patients suffering from SMN1-associated infantile form of the disease with a splice-switching antisense oligonucleotide (ASO) that corrects aberrant splicing of the nearly identical SMN2 gene to allow the generation of functional SMN protein, thereby mitigating the development of the disease...
June 22, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28622288/optimization-of-design-and-production-strategies-for-novel-adeno-associated-viral-display-peptide-libraries
#10
J K├Ârbelin, A Hunger, M Alawi, T Sieber, M Binder, M Trepel
Libraries displaying random peptides on the surface of adeno-associated virus (AAV) are powerful tools for the generation of target-specific gene therapy vectors. However, for unknown reasons the success rate of AAV library screenings is variable and the influence of the production procedure has not been thoroughly evaluated. During library screenings, the capsid variants with the most favorable tropism are enriched over several selection rounds on a target of choice and identified by subsequent sequencing of the encapsidated viral genomes encoding the library capsids with targeting peptide insertions...
June 16, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28617420/mirna-mediated-post-transcriptional-silencing-of-transgenes-leads-to-increased-adeno-associated-viral-vector-yield-and-targeting-specificity
#11
C A Reid, S L Boye, W W Hauswirth, D M Lipinski
The production of high titer recombinant adeno-associated virus (rAAV) vector is essential for treatment of genetic diseases affecting the retina and choroid, where anatomical constraints may limit injectable volumes. Problematically, cytotoxicity arising from over-expression of the transgene during vector production frequently leads to a reduction in vector yield. Herein, we evaluate the use of micro-RNA (miRNA) mediated silencing to limit over-expression of cytotoxic transgenes during packaging as a method of increasing vector yield...
June 15, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28561814/the-sma-trust-the-role-of-a-disease-focused-research-charity-in-developing-treatments-for-sma
#12
REVIEW
V Christie-Brown, J Mitchell, K Talbot
SMA is a rare hereditary neuromuscular disease that causes weakness and muscle wasting as a result of the loss of spinal motor neurons. In its most severe form, SMA is the commonest genetic cause of death in infants, and children with less severe forms of SMA face the prospect of lifelong disability from progressive muscle wasting, loss of mobility and limb weakness. The initial discovery of the defective gene has been followed by major advances in our understanding of the genetic, cellular and molecular basis of SMA, providing the foundation for a range of approaches to treatment, including gene therapy, antisense oligonucleotide treatments and more traditional drug-based approaches to slow or halt disease progression...
May 31, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28561813/therapeutic-approaches-for-spinal-muscular-atrophy-sma
#13
REVIEW
M Scoto, R S Finkel, E Mercuri, F Muntoni
Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder characterized by progressive muscle wasting and loss of muscle function due to severe motor neuron dysfunction, secondary to mutations in the survival motor neuron 1 (SMN1) gene. A second neighboring centromeric gene, SMN2, is intact in all patients but contains a C-to-T variation in exon 7 that affects a splice enhancer and determines exclusion of exon 7 in the majority of its transcript, leading to an unstable protein that cannot substitute for mutant SMN1...
May 31, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28556834/developmental-regulation-of-smn-expression-pathophysiological-implications-and-perspectives-for-therapy-development-in-spinal-muscular-atrophy
#14
REVIEW
S Jablonka, M Sendtner
Spinal muscular atrophy (SMA), the predominant form of motoneuron diease in children and young adults is caused by loss of function of the SMN protein. On the basis of a disrupted splice acceptor site in exon 7, transcripts from a second SMN gene in humans called SMN2 cannot give rise to SMN protein at sufficient levels for maintaining function of motoneurons and motor circuits. First clinical trials with Spinraza/Nusinersen, a drug that counteracts disrupted splicing of SMN2 transcripts, have shown that elevating SMN levels can successfully interfere with motoneuron dysfunction...
May 30, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28553929/silencing-of-hiv-1-by-agoshrna-molecules
#15
E Herrera-Carrillo, A Harwig, B Berkhout
RNA interference (RNAi) is a sequence-specific gene silencing mechanism that is triggered by the expression of a short hairpin RNA (shRNA). shRNA molecules enter the RNAi pathway at the Dicer processing step. Recent studies indicated that the cellular microRNA (miRNA) miR-451 is not recognized by Dicer, but that is processed instead by the Argonaute 2 (Ago2) protein. Subsequently, Dicer-independent shRNAs were described that rely on Ago2 for processing as well as the subsequent silencing step. We called these AgoshRNA molecules because they depend on Ago2 both for maturation and activation...
May 29, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28504658/cure-sma-and-our-patient-community-celebrate-the-first-approved-drug-for-sma
#16
REVIEW
J Glascock, M Lenz, K Hobby, J Jarecki
Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA)-a disease affecting motor neurons, that robs patients of their ability to walk, eat and even breathe. Since 1984, we have directed and invested in comprehensive research that has shaped the scientific community's understanding of SMA. On 23 December, 2016, the Food and Drug Administration (FDA) announced approval of Spinraza, a treatment developed by Biogen and Ionis, making it the first-ever approved therapy for SMA. Cure SMA provided early research funding in 2003 leading to the discovery of ISS-N1 sequence, now targeted by Spinraza...
May 15, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28504657/engineering-liposomal-nanoparticles-for-targeted-gene-therapy
#17
REVIEW
C Zylberberg, K Gaskill, S Pasley, S Matosevic
Recent mechanistic studies have attempted to deepen our understanding of the process by which liposome-mediated delivery of genetic material occurs. Understanding the interactions between lipid nanoparticles and cells is still largely elusive. Liposome-mediated delivery of genetic material faces systemic obstacles alongside entry into the cell, endosomal escape, lysosomal degradation and nuclear uptake. Rational design approaches for targeted delivery have been developed to reduce off-target effects and enhance transfection...
May 15, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28467403/building-the-patient-community
#18
REVIEW
F Raffai, O Timmis
There are many challenges in conducting rare disease research. The conditions are often poorly understood, small patient populations are dispersed around the world, and there are limited funding opportunities. Patient groups can serve as a key partner in overcoming these challenges, as they understand the impact of rare conditions on patients' lives. This gives patient groups valuable scientific insights into the disease. This can be used to create research strategies, address research bottlenecks and directly fund research that appropriately addresses patient needs...
May 11, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28467402/new-treatments-for-serious-conditions-ethical-implications
#19
REVIEW
N M P King, C E Bishop
Approval of Spinraza (nusinersen) for treatment of spinal muscular atrophy prompts consideration of a number of ethical issues that arise whenever a new treatment is proposed for a serious condition, especially one that is rare and can devastatingly affect children. Patients, families, clinicians, researchers, institutions and policymakers all must take account of the ways that newly available treatments affect informed and shared decision-making about therapeutic and research options. The issues to consider include: addressing what is still uncertain and unknown; the possibility that potential benefits will be exaggerated and potential harms underemphasized in the media, by advocacy organizations, and in consent forms and processes; the high cost of many novel drugs and biologics; the effects of including conditions of variable phenotype in state-mandated newborn screening panels; and how new treatments can change the standard of care, altering what is and is not known about a disorder and posing challenges for decision-making at both individual and policy levels...
May 11, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28485723/developing-gene-and-cell-therapies-for-rare-diseases-an-opportunity-for-synergy-between-academia-and-industry
#20
REVIEW
F Mavilio
For the last twenty years, academic research has been the major, and often only, driving force behind the spectacular development of gene transfer technology for the therapy of rare genetic diseases. Investors and industry became eventually interested in gene and cell therapy, due to the success of a series of pioneering clinical trials that proved efficacy and safety of last-generation technology, and to favorable orphan drug legislation in both Europe and the United States. Developing this forms of therapy is however complex and requires skills and knowledge not necessary available to the industry, which is better placed to develop processes and products and put them on the market...
May 9, 2017: Gene Therapy
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