Peptide Shuttle-Mediated Delivery for Brain Gene Therapies

The manipulation of an individual’s genetic information to treat a disease has revolutionized the biomedicine field. Despite the promise of gene therapy, this treatment can have long-term sideeffects. Efforts in the field and recent discoveries have already led to several improvements, including efficient gene delivery and transfer, as well as inpatient safety. Several studies to treat a wide range of pathologies-such as cancer or monogenic diseases- are currently being conducted. Here we provide a broad overview of methodologies available for gene therapy, placing a strong emphasis on treatments for central nervous system diseases. Finally, we give a perspective on current delivery strategies to treat such diseases, with a special focus on systems that use peptides as delivery vectors.

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The Promise and the Hope of Gene Therapy

Frontiers in Genome Editing ◽

10.3389/fgeed.2021.618346 ◽

2021 ◽

Vol 3 ◽

Author(s):

Eleni Papanikolaou ◽

Andreas Bosio

Keyword(s):

Gene Therapy ◽

Hemopoietic Stem Cell ◽

Cell Therapies ◽

Gene Therapies ◽

Monogenic Diseases ◽

Car T ◽

The Everyday ◽

Cell Gene ◽

Stem Cell Gene Therapy

It has been over 30 years since visionary scientists came up with the term “Gene Therapy,” suggesting that for certain indications, mostly monogenic diseases, substitution of the missing or mutated gene with the normal allele via gene addition could provide long-lasting therapeutic effect to the affected patients and consequently improve their quality of life. This notion has recently become a reality for certain diseases such as hemoglobinopathies and immunodeficiencies and other monogenic diseases. However, the therapeutic wave of gene therapies was not only applied in this context but was more broadly employed to treat cancer with the advent of CAR-T cell therapies. This review will summarize the gradual advent of gene therapies from bench to bedside with a main focus on hemopoietic stem cell gene therapy and genome editing and will provide some useful insights into the future of genetic therapies and their gradual integration in the everyday clinical practice.

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Advanced Materials for Gene Delivery

Advanced Materials Research ◽

10.4028/www.scientific.net/amr.995.29 ◽

2014 ◽

Vol 995 ◽

pp. 29-47 ◽

Cited By ~ 1

Author(s):

Mohammad A. Jafar Mazumder ◽

Md. Hasan Zahir ◽

Sharif F. Zaman

Keyword(s):

Gene Therapy ◽

Gene Delivery ◽

Genetic Disorders ◽

Basic Research ◽

Advanced Materials ◽

Clinical Settings ◽

Efficient Gene ◽

Efficient Delivery ◽

Promising Treatment

Gene therapy is a widespread and promising treatment of many diseases resulting from genetic disorders, infections and cancer. The feasibility of the gene therapy is mainly depends on the development of appropriate method and suitable vectors. For an efficient gene delivery, it is very important to use a carrier that is easy to produce, stable, non-oncogenic and non-immunogenic. Currently most of the vectors actually suffer from many problems. Therefore, the ideal gene therapy delivery system should be developed that can be easily used for highly efficient delivery and able to maintain long-term gene expression, and can be applicable to basic research as well as clinical settings. This article provides a brief over view on the concept and aim of gene delivery, the different gene delivery systems and use of different materials as a carrier in the area of gene therapy.

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Artificial miRNAs are potential gene therapy tools, especially for incurable monogenic disorders

10.31219/osf.io/d5rnm ◽

2021 ◽

Author(s):

Moataz Dowaidar

Keyword(s):

Gene Therapy ◽

Brain Surgery ◽

Native Protein ◽

Intravenous Injections ◽

Monogenic Disorders ◽

Monogenic Diseases ◽

Short Hairpin ◽

The Cost ◽

Short Hairpin Rnas

Well-designed artificial miRNAs (amiRNAs) are as effective as short hairpin RNAs (shRNAs) but produce 10–80 times less siRNA. They enable long-term silencing and are safer than other RNAi triggers. They are suitable instruments for gene therapy techniques, especially for incurable monogenic diseases. In clinical studies, stereotactic injection of AAV5 directly into the striatum is the most effective approach. Intravenous injections would not only make patients more comfortable, but would also reduce the cost of complex brain surgery. In terms of structure, biogenesis, and expression levels, Ami RNAs are more "natural" than other gene therapy methods. They also utilise the cell's native protein machinery and do not produce irreversible alterations, unlike genome editing technologies. The amount of time spent on a technology determines its level of progression. ASOs have an edge in this regard, as seen by the number of authorized medicines. Perhaps RNAi is just around the corner.

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Gene Therapy for Infectious Diseases

Clinical Microbiology Reviews ◽

10.1128/cmr.11.1.42 ◽

1998 ◽

Vol 11 (1) ◽

pp. 42-56 ◽

Cited By ~ 65

Author(s):

Bruce A. Bunnell ◽

Richard A. Morgan

Keyword(s):

Gene Therapy ◽

Infectious Diseases ◽

Infectious Agent ◽

Catalytic Rna ◽

Single Chain ◽

Gene Therapies ◽

Single Chain Antibodies ◽

Wide Range ◽

Management Approaches ◽

I Gene

SUMMARY Gene therapy is being investigated as an alternative treatment for a wide range of infectious diseases that are not amenable to standard clinical management. Approaches to gene therapy for infectious diseases can be divided into three broad categories: (i) gene therapies based on nucleic acid moieties, including antisense DNA or RNA, RNA decoys, and catalytic RNA moieties (ribozymes); (ii) protein approaches such as transdominant negative proteins and single-chain antibodies; and (iii) immunotherapeutic approaches involving genetic vaccines or pathogen-specific lymphocytes. It is further possible that combinations of the aforementioned approaches will be used simultaneously to inhibit multiple stages of the life cycle of the infectious agent.

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Retroviral gene therapy in Germany with a view on previous experience and future perspectives

Gene Therapy ◽

10.1038/s41434-021-00237-x ◽

2021 ◽

Author(s):

Michael A. Morgan ◽

Melanie Galla ◽

Manuel Grez ◽

Boris Fehse ◽

Axel Schambach

Keyword(s):

Gene Therapy ◽

Cell Function ◽

Immune Cell ◽

Retroviral Vectors ◽

Hematopoietic Stem ◽

Gene Therapy Vector ◽

Cell Therapies ◽

Gene Therapies ◽

Monogenic Disorders ◽

Monogenic Diseases

AbstractGene therapy can be used to restore cell function in monogenic disorders or to endow cells with new capabilities, such as improved killing of cancer cells, expression of suicide genes for controlled elimination of cell populations, or protection against chemotherapy or viral infection. While gene therapies were originally most often used to treat monogenic diseases and to improve hematopoietic stem cell transplantation outcome, the advent of genetically modified immune cell therapies, such as chimeric antigen receptor modified T cells, has contributed to the increased numbers of patients treated with gene and cell therapies. The advancement of gene therapy with integrating retroviral vectors continues to depend upon world-wide efforts. As the topic of this special issue is “Spotlight on Germany,” the goal of this review is to provide an overview of contributions to this field made by German clinical and research institutions. Research groups in Germany made, and continue to make, important contributions to the development of gene therapy, including design of vectors and transduction protocols for improved cell modification, methods to assess gene therapy vector efficacy and safety (e.g., clonal imbalance, insertion sites), as well as in the design and conduction of clinical gene therapy trials.

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Modern prevention and active longevity programs on the basis of disruptive domestic technologies: positive experience and prospects for application

Terapevt (General Physician) ◽

10.33920/med-12-2001-09 ◽

2020 ◽

pp. 66-73

Author(s):

A. Simonova ◽

S. Chudakov ◽

R. Gorenkov ◽

V. Egorov ◽

A. Gostry ◽

...

Keyword(s):

Personalized Medicine ◽

Early Detection ◽

Laboratory Diagnostics ◽

Positive Experience ◽

Practical Application ◽

Wide Range ◽

Integrated Program ◽

Long Term Experience ◽

Early Detection And Prevention

The article summarizes the long-term experience of practical application of domestic breakthrough technologies of preventive personalized medicine for laboratory diagnostics of a wide range of socially significant non-infectious diseases. Conceptual approaches to the formation of an integrated program for early detection and prevention of civilization diseases based on these technologies are given. A vision of the prospects for the development of this area in domestic and foreign medicine has been formed.

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