Concepts in genetic medicine

TL;DR: Steps involved in the cell processing of the technology, including the use of an optimal vector for consistent cell processing, are discussed, along with addressing the challenges of expanding chimeric antigen receptor T cell therapy to a global patient population.

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637

•Journal Article

Inhibition of hepatitis B virus in mice by RNA interference

A. P. McCaffrey

- 01 Jan 2005

- Nature Medicine

TL;DR: It is shown that RNAi can be applied to inhibit production of HBV replicative intermediates in cell culture and in immunocompetent and immunodeficient mice transfected with an HBV plasmid, showing that such an approach could be useful in the treatment of viral diseases.

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401

Journal Article•10.1517/14712598.8.4.475

T lymphocyte engineering ex vivo for cancer and infectious disease.

Bruce L. Levine

- 19 Mar 2008

- Expert Opinion on Biological Therapy

TL;DR: Advances in the technological approaches and methods for ex vivo manipulation have led to T lymphocytes endowed with enhanced potency and unique functions, with promise as the new generation of infused therapeutics.

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35

Highly efficient EIAV-mediated in utero gene transfer and expression in the major muscle groups affected by Duchenne Muscular Dystrophy.

Lisa G. Gregory, +13 more

- 01 Jan 2004

TL;DR: In this article, the authors applied s-galactosidase expressing equine infectious anaemia virus (EIAV) pseudotyped with VSV-G by single or combined injection via different routes to the MF1 mouse fetus on day 15 of gestation and describe substantial gene delivery to the musculature.

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References

Journal Article•10.1093/JNCI/97.9.626

Blocking Cancer With RNA Interference Moves Toward the Clinic

Karyn Hede

- 04 May 2005

- Journal of the National Cancer Institute

TL;DR: In back-to-back papers published in the February issue of Nature Biotechnology, John Rossi and Hannon’s group described how to design siRNA templates that effectively activate a group of proteins called the RNAinduced silencing complex (RISC), whose activation results in sequence-specifi c gene silencing.

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28

•Journal Article•10.1016/J.YMTHE.2004.03.019

Transduction of Cell Lines and Primary Cells by FIV-Packaged HIV Vectors

Kevin V. Morris, +4 more

- 01 Jul 2004

- Molecular Therapy

TL;DR: It is demonstrated that packaging HIV vectors with FIV is a viable alternative, which avoids use of HIV structural proteins, and to remove the possibility of seroconversion to HIV proteins.

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28

Journal Article•10.2174/1566523043346174

Safety concerns related to hematopoietic stem cell gene transfer using retroviral vectors.

Peter Haviernik, +1 more

- 31 Aug 2004

- Current Gene Therapy

TL;DR: In the field of gene therapy, retroviral vector design and safety testing are at the forefront of the field as discussed by the authors, with the goal of gene transfer into hematopoietic stem cells (HSCs).

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28

Journal Article•10.1016/S0165-6147(99)01350-4

Gene therapy for lung disease

David L. Ennist

- 01 Jun 1999

- Trends in Pharmacological Sciences

TL;DR: Gene therapy is now beginning to benefit from a shift in emphasis from clinical trials to the development of better tools and procedures to deliver gene therapy to the bedside, and is focusing on further research and technological improvements to the vectors.

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