Gene therapy finds its niche

  title={Gene therapy finds its niche},
  author={Cormac Sheridan},
  journal={Nature Biotechnology},
  • C. Sheridan
  • Published 7 February 2011
  • Medicine
  • Nature Biotechnology
Gene therapy is finally poised to make a contribution to the treatment of debilitating, highly penetrant genetic diseases that have proved intractable to other regimens. 

Gene therapy on the move 1642 Gene therapy on the move

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State-of-the-art gene-based therapies: the road ahead

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  • 2011
Improvements in the gene transfer vectors used in therapeutic trials have led to substantial clinical successes in patients with serious genetic conditions, such as immunodeficiency syndromes,

Applications of Gene Editing Technologies to Cellular Therapies.

Recent Progresses in Brain Gene Therapy

This review tried to focus on techniques and approaches in brain tumors and nervous system’s gene therapy strategies and will discuss about associated problems and potential future in management of inherited or acquired neurological disorders.

Cell-Based Gene Therapy

Three critical aspects have emerged for all gene transfer approaches which are also fundamental to further develop this novel and highly sophisticated therapy.

Cell type specific gene delivery by lentiviral vectors

Recent findings about a CD8-specific vector that enhances the killing of tumor cells in TCR-based gene transfer strategies are explained.

Gene Therapy in the Management of Head and Neck Cancers: A Review. -

Different treatment approaches are revealed for cancer gene therapy; gene addition therapy, suicide gene therapy, immunotherapy, and different types of gene therapies are discussed along with an insight into future of this treatment modality.



20 years of gene therapy for SCID

The past 20 years has shown that severe combined immunodeficiency conditions are correctable by gene therapy, and these diseases were initially treated by transplantation of allogeneic hematopoietic stem cells.

Gene therapy: cursed or inching towards credibility?

A chastened but determined group of pioneers believes gene therapy can be lived down and live up to its initial promise, and they are pointing to a new generation of products to back up that claim.

T Lymphocyte-Directed Gene Therapy for ADA− SCID: Initial Trial Results After 4 Years

It is concluded here that gene therapy can be a safe and effective addition to treatment for some patients with this severe immunodeficiency disease.

Splicing Out the West?

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Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

Gene therapy, combined with reduced-intensity conditioning, is a safe and effective treatment for SCID in patients with ADA deficiency and effective protection against infections and improvement in physical development made a normal lifestyle possible.

A brief history of gene therapy

Efficient retroviral vectors and other gene transfer methods have permitted convincing demonstrations of efficient phenotype correction in vitro and in vivo, now making gene therapy a broadly accepted approach to therapy and justifying clinically applied studies with human patients.

Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.

A gene therapy trial for SCID-X1 was initiated, based on the use of complementary DNA containing a defective gammac Moloney retrovirus-derived vector and ex vivo infection of CD34+ cells, which provided full correction of disease phenotype and clinical benefit.

Toward gene therapy for cystic fibrosis using a lentivirus pseudotyped with Sendai virus envelopes.

The data suggest that this lentiviral vector may provide a step change in airway transduction efficiency relevant to a clinical programme of gene therapy for CF.

CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression

Using a CpG-free pDNA expression vector, sustained in vivo transgene expression is achieved in the absence of lung inflammation and it is demonstrated that retention of even a single CPG in pDNA is sufficient to elicit an inflammatory response, whereas C pG- free pDNA vectors do not.

Gene Therapy for Human Genetic Disease?

A sustained effort is proposed to be made to formulate a complete set of ethicoscientific criteria to guide the development and clinical application of gene therapy techniques, which could go a long way toward ensuring that gene therapy is used in humans only in those instances where it will prove beneficial, and toward preventing its misuse through premature application.