scholarly journals Challenging times: Delivering gene therapies and an opportunity for shared learning

2021 ◽  
Author(s):  
Cormac Kennedy ◽  
Edel O'Dea ◽  
Derval Reidy ◽  
Jeremy Towns ◽  
Maria Pigott ◽  
...  
Keyword(s):  
Shared Learning ◽  
Gene Therapies

Nanostructured Hyaluronic Acid-based Materials for the Delivery of siRNA

2018 ◽  
Vol 24 (23) ◽  
pp. 2678-2691 ◽  
Author(s):  
Keval Shah ◽  
Sunita Chawla ◽  
Anuradha Gadeval ◽  
Goutham Reddy ◽  
Rahul Maheshwari ◽  
...  
Keyword(s):  
Hyaluronic Acid ◽  
Rna Interference ◽  
Sirna Delivery ◽  
Treatment Strategies ◽  
Aqueous Solubility ◽  
Micro Rna ◽  
Target Space ◽  
Significant Progress ◽  
Gene Therapies ◽  
Future Potential

Background: The search for the effective treatment strategies to combat a disease that is characterized by abnormal cell growth and known as cancer is still required to reach its destiny. To address the problem, recently several gene therapies based on novel RNA interference (RNAi) have been proposed such as siRNA, micro RNA, shRNA, etc. out of which, siRNAs (silencing RNA) promises to show significant progress in pharmacotherapy, including considerable expansion of the druggable target space and the possibility of treating cancer. Methods: This review aims to uncover the hyaluronic acid (HA) and HA-hybridized nanoplatforms for siRNA delivery systems with a particular focus on the discussion of available reports while addressing the future potential of HA-based treatment strategies. Results: HA modified siRNA delivery, as promised, provided better targeting potential in many types of cancers. In addition, it was able to modify the release of siRNA as well. Toxicity of HA is well mentioned however, the loophole is yet to be filled by exploring various remedies for overcoming toxicity. Conclusion: To overcome the problems associated with these emerging genetic tools, investigators have employed glycosaminoglycan HA-based biopolymers. This biopolymer offers a variety of properties such as biodegradability, biocompatibility, aqueous solubility, viscoelasticity, and non-immunogenicity.

Huntington Disease

2019 ◽  
pp. 644-678
Author(s):  
Jane S. Paulsen
Keyword(s):  
Huntington Disease ◽  
Trinucleotide Repeat ◽  
Behavioral Control ◽  
Time Estimation ◽  
Brain Dysfunction ◽  
Medium Spiny Neuron ◽  
Loss Of Function ◽  
Neuron Death ◽  
Gene Therapies ◽  
Underlying Mechanisms

Huntington disease (HD) is a autosomal dominant neurodegenerative disease caused by expansion of a trinucleotide repeat (cytosine, adenine, and guanine [CAG]) on the short arm of chromosome four. Average age of motor diagnosis is 39 years, and age at diagnosis is associated with the length of the CAG mutation. The prodrome of HD can be recognized 15 years prior to motor diagnosis and is characterized by subtle impairments in emotional recognition, smell identification, speed of processing, time estimation and production, and psychiatric abnormalities. HD shows particular vulnerability of the medium spiny neuron in the basal ganglia. Progressive brain dysfunction and neuron death lead to insidious loss of function in motor, cognitive, and behavioral control over 34 years (17 prodromal and 17 post-diagnosis). Treatment plans rely on genetic counseling, psychiatric symptom treatment as needed, physical therapy, and environmental modifications. There are two treatments for the reduction of chorea, but there are no disease-modifying therapies. Experimental therapeutics are rapidly emerging with multiple and various targets, however, and gene therapies to silence the mutant HD gene are currently ongoing. This chapter reviews clinical and neuropathological descriptions of HD and discusses potential underlying mechanisms and animal models, diagnostic and clinical assessments used to characterize and track the disease, treatment planning, and challenges for research to advance care.

Gene Therapies for Transfusion-Dependent β-Thalassemia

2021 ◽  
Vol 58 (7) ◽  
pp. 667-674
Author(s):  
Sandeep Soni
Keyword(s):  
Gene Therapies

scholarly journals Automatic 1D 1H NMR Metabolite Quantification for Bioreactor Monitoring

Metabolites ◽  
2021 ◽  
Vol 11 (3) ◽  
pp. 157
Author(s):  
Roy Chih Chung Wang ◽  
David A. Campbell ◽  
James R. Green ◽  
Miroslava Čuperlović-Culf
Keyword(s):  
High Throughput ◽  
Nmr Metabolomics ◽  
Gene Therapies ◽  
Quantification Method ◽  
Minimal Sample ◽  
Enhanced Production ◽  
Bioreactor Monitoring ◽  
Online Measurements ◽  
Metabolite Quantification ◽  
Set Up

High-throughput metabolomics can be used to optimize cell growth for enhanced production or for monitoring cell health in bioreactors. It has applications in cell and gene therapies, vaccines, biologics, and bioprocessing. NMR metabolomics is a method that allows for fast and reliable experimentation, requires only minimal sample preparation, and can be set up to take online measurements of cell media for bioreactor monitoring. This type of application requires a fully automated metabolite quantification method that can be linked with high-throughput measurements. In this review, we discuss the quantifier requirements in this type of application, the existing methods for NMR metabolomics quantification, and the performance of three existing quantifiers in the context of NMR metabolomics for bioreactor monitoring.

Trojan horse at cellular level for tumor gene therapies

Gene ◽  
2013 ◽  
Vol 525 (2) ◽  
pp. 208-216 ◽  
Author(s):  
Guillaume Collet ◽  
Catherine Grillon ◽  
Mahdi Nadim ◽  
Claudine Kieda
Keyword(s):  
Cellular Level ◽  
Trojan Horse ◽  
Gene Therapies ◽  
Tumor Gene

scholarly journals Clinical Perspectives and Trends: Microperimetry as a trial endpoint in retinal disease

2021 ◽  
Author(s):  
Yesa Yang ◽  
Hannah Dunbar
Keyword(s):  
Clinical Trial ◽  
Retinal Disease ◽  
Age Related Macular Degeneration ◽  
Gene Therapies ◽  
Age Related ◽  
Wide Range ◽  
Trial Endpoints ◽  
Visual Acuity Loss ◽  
Psychophysical Test ◽  
Trial Endpoint

Endpoint development trials are underway across the spectrum of retinal disease. New validated endpoints are urgently required for the assessment of emerging gene therapies and in preparation for the arrival of novel therapeutics targeting early stages of common sight-threatening conditions such as age-related macular degeneration. Visual function measures are likely to be key candidates in this search. Over the last two decades, microperimetry has been used extensively to characterize functional vision in a wide range of retinal conditions, detecting subtle defects in retinal sensitivity that precede visual acuity loss and tracking disease progression over relatively short periods. Given these appealing features, microperimetry has already been adopted as an endpoint in interventional studies, including multicenter trials, on a modest scale. A review of its use to date shows a concurrent lack of consensus in test strategy and a wealth of innovative disease and treatment-specific metrics which may show promise as clinical trial endpoints. There are practical issues to consider, but these have not held back its popularity and it remains a widely used psychophysical test in research. Endpoint development trials will undoubtedly be key in understanding the validity of microperimetry as a clinical trial endpoint, but existing signs are promising.

A plethora of gene therapies for hemoglobinopathies

2021 ◽  
Vol 27 (2) ◽  
pp. 202-204
Author(s):  
Cynthia E. Dunbar
Keyword(s):  
Gene Therapies
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