A Brief History of Genetic Therapy: Gene Therapy, Antisense Technology, and Genomics

2020 ◽  
pp. 1-38
Author(s):  
James W. Hawkins
Keyword(s):  
Gene Therapy ◽  
Genetic Therapy ◽  
Antisense Technology ◽  
History Of

scholarly journals The Ocular Gene Delivery Landscape

Biomolecules ◽  
2021 ◽  
Vol 11 (8) ◽  
pp. 1135
Author(s):  
Bhubanananda Sahu ◽  
Isha Chug ◽  
Hemant Khanna
Keyword(s):  
Gene Therapy ◽  
Genetic Diseases ◽  
Gene Therapies ◽  
Advantages And Disadvantages ◽  
Fda Approval ◽  
New Information ◽  
Efficient Delivery ◽  
Delivery Routes ◽  
History Of ◽  
Different Parts

The eye is at the forefront of developing therapies for genetic diseases. With the FDA approval of the first gene-therapy drug for a form of congenital blindness, numerous studies have been initiated to develop gene therapies for other forms of eye diseases. These examinations have revealed new information about the benefits as well as restrictions to using drug-delivery routes to the different parts of the eye. In this article, we will discuss a brief history of gene therapy and its importance to the eye and ocular delivery landscape that is currently being investigated, and provide insights into their advantages and disadvantages. Efficient delivery routes and vehicle are crucial for an effective, safe, and longer-lasting therapy.

scholarly journals GENE THERAPY FOR HAEMOPHILIA

2021 ◽  
Vol 2021 (1) ◽  
Author(s):  
A Asghar ◽  
Z Asjad ◽  
H Tahir ◽  
Z Maheen ◽  
S Hanif
Keyword(s):  
Gene Therapy ◽  
Gene Editing ◽  
Genetic Disorders ◽  
Coagulation Factor ◽  
Retroviral Vectors ◽  
Adeno Associated Virus ◽  
General Application ◽  
Blood Disorder ◽  
Statistical Consideration ◽  
History Of

The blood disorder, Hemophilia, has its roots embedded deep into the history of genetic disorders. The European royal family is one of the most prominent families to be affected by this disease thus, dubbing it 'the royal disease'. The types of Hemophilia are divided into two based on the type of coagulation factor mutation found in the patient. For treating haemophilia, gene therapy is done by using different vectors such as lentiviral and retroviral vectors but due to the production of limited expression different adeno associated virus (AAV) strains are used. Some engineerly modified vectors are currently used to get the best possible results. The clinical trials prove the efficacy of these vectors so through their obtained statistical consideration, patient experience and population study once can design vaccines and drugs for haemophilia patients but also due to pre-existing Nabs and pre-existing HCV or HBV infection, the general application of AAV gene therapy is currently limited. The possibility of gene editing for the repair of the mutation is on the horizon.

Discoveries in gene-environment interactions that influence CVD, lipid traits, obesity, diabetes, and hypertension appear to be able to influence gene therapy.

2021 ◽  
Author(s):  
Moataz Dowaidar
Keyword(s):  
Gene Therapy ◽  
Genetic Risk ◽  
Environmental Influences ◽  
Biological Processes ◽  
Significant Progress ◽  
Therapy Outcomes ◽  
Genetic Therapy ◽  
Clinical Genetic ◽  
Gene Environment ◽  
Made In

In a relatively short amount of time, significant progress has been made in discovering gene-environment interactions that influence CVD, lipid traits, obesity, diabetes, and hypertension. These correlations appear to change genetic vulnerability, which may help researchers better understand the genetic processes that influence CVD development in the future. In order to advance the field, further research is required to confirm initial comparisons, identify the biological processes by which environmental influences modify genetic risk, and investigate strategies that use this knowledge to influence clinical genetic therapy outcomes.

scholarly journals Mild SARS-CoV-2 Infection After Gene Therapy in a Child With Wiskott-Aldrich Syndrome: A Case Report

2020 ◽  
Vol 11 ◽  
Author(s):  
Sabina Cenciarelli ◽  
Valeria Calbi ◽  
Federica Barzaghi ◽  
Maria Ester Bernardo ◽  
Chiara Oltolini ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Case Report ◽  
Immune Reconstitution ◽  
Autoinflammatory Disease ◽  
Clinical Phenotype ◽  
High Titer ◽  
Previous History ◽  
Wiskott Aldrich Syndrome ◽  
Neutralizing Capacity ◽  
History Of

In this work we present the case of SARS-CoV-2 infection in a 1.5-year-old boy affected by severe Wiskott-Aldrich Syndrome with previous history of autoinflammatory disease, occurring 5 months after treatment with gene therapy. Before SARS-CoV-2 infection, the patient had obtained engraftment of gene corrected cells, resulting in WASP expression restoration and early immune reconstitution. The patient produced specific immunoglobulins to SARS-CoV-2 at high titer with neutralizing capacity and experienced a mild course of infection, with limited inflammatory complications, despite pre-gene therapy clinical phenotype.

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