International Conference on Genetic Disorders and Gene Therapy May 18-19, 2020 Auckland, Newzealand

A number of methods are under development for treating genetic disorders, including a inherited (eg, monogenic/Mendelian) conditions and acquired conditions such as cancer and infections. This topic reviews molecular techniques that can be used to alter the sequence or expression of a gene, including gene therapy, gene editing, and gene silencing.

Stem cell treatment is a process where the stem cells are used to make disease free of life through treatment of diseases. They have the potential to treat various life threatening diseases. The unique feature of these cells to divide into multiple stem cells and differentiate into specialized cells makes them apt for the treatment of many diseases. The present researches suggest that stem cells can be used for treating the genetic disorders as well. Apart from treating diseases like brain injuries, Osteoarthritis, diabetes,  learning disabilities, various types of blood cancer, Sickle cell anemia, Parkinson’s, Haemophilia, and Thalassemia, etc.., The cell defect due to mutation result in a wide of variety of genetic disorders. The transplantation of stem cells where the normal stem cell replaces the defected cells can be used for treatment of genetic disorders and diseases.

Most children are born healthy with no medical problems or birth defects. However, some children are born with differences in body structure, brain development, or body chemistry that can lead to problems with health, development, school performance, and/or social interaction. Pediatric geneticists are trained to identify the causes and natural history of these disorders. They may suggest tests and treatments that can help in understanding and caring for your child’s condition. Pediatric geneticists also can help families understand whether some conditions are hereditary (coming from the genes) and offer testing to family members who may be at risk for having children with similar problems.

Gene therapy essentially involves the introduction or alteration of genetic material among the cell or organism with associate intention of curing of the sickness. The individual cell therapy and gene therapy uses overlapping fields of medical analysis with the goals of repairing the direct  Genetic diseases in polymer or cellular population respectively, the invention of DNA technology within the 1970’s provided tools with efficiency develop gene theraphy. Scientists use these techniques to promptly manipulate infective agent genomes, isolate genes and establish mutations concerned in human sickness, characterize and regulate sequence expressions, and engineer numerous infective agent and non-viral vectors. Numerous long-term treatments for anemia, hemophilia, mucoviscidosis, genetic defect, Gauscher’s sickness, lysosomal storage diseases, vas diseases, polygenic disease and diseases of bones and joints are resolved through successful gene therapy.

A genetic disorder is a disease caused in whole or in part by a change in the DNA sequence away from the normal sequence. Genetic disorders can be caused by a mutation in one gene (monogenic disorder), by mutations in multiple genes (multifactorial inheritance disorder), by a combination of gene mutations and environmental factors, or by damage to chromosomes (changes in the number or structure of entire chromosomes, the structures that carry genes).

Genome editing with engineered nucleases (GEEN) is emergent type of Genetic Engineering. GEEN is the technology in which DNA is inserted, deleted or replaced in the genome. The emergence of highly versatile genome-editing technologies has provided investigators with the ability to rapidly and economically introduce sequence-specific modifications into the genomes of a broad spectrum of cell types and organisms. It also promotes various changes in sub cellular level. Genome Editing itself also holds tremendous potential for treating the underlying various idiopathic genetic causes of certain diseases. The core technologies now most commonly used techniques to facilitate genome editing are clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), transcription activator-like effector nucleases (TALENs), zinc-finger nucleases (ZFNs), and homing endonucleases or mega nucleases.

Diseases can be treated using viruses as vector to deliver genes in Gene Therapy. Viruses as gene vector, however, can themselves cause problems in that they may initiate inflammation and the genes may be expressed at too high a level or for too long period of exposure. The goal of Nano Technology in gene therapy is delivery of therapeutic genes without a virus, using nanoparticles as non-viral vector to deliver the genes.  The particles can be made with multiple layers so the outer layer with covering of peptide that can target the particles to cells of interest at specific site. The emergent Nanotechnology in gene therapy is used to develop unique approaches in treating the retinopathies and the development of micro and Nano dimensional artificial antigen presenting cells (aAPCs) for cancer immunotherapy. These aAPCs mimic the natural signals in immunity that killer T-cells receive when there is an invader (bacteria, virus, cancer cell, etc.) in the body.

Clinical Trials of Cell and gene therapy product usually variable from the clinical trials design for alternative varieties of pharmaceutical product. This variations in trial design that are necessitated by the distinctive options of those product. The clinical trials additionally replicate previous clinical expertise and proof of drugs. Early experiences with Cell and gene therapy product indicate that some CGT product might cause substantial risks to subjects because of impact at cellular and genetic level. The planning of early-phase clinical trials of Cell and gene therapy product usually involves the subsequent thought of clinical questions of safety, diagnosing problems, and chemistry, producing and controls (CMC) problems that can be encountered.

Biomarkers have rapid evolvement in the advance of personalized medicine and individual health Biomarkers refers to a broad subcategory of medical signs which include objective indications of medical state that are determined from outside the patient – which might be measured accurately and reproducibly. Medical signs change distinction to medical symptoms, that are restricted to those indications of health or sickness perceived by patients themselves.

Cell therapy products require a variety of safety considerations. Stem cell and gene products are heterogeneous substances. There are several areas that particularly need to be addressed as it is quite different from that of pharmaceuticals. These range from creating batch consistency, product stability to product safety, strength and efficacy through pre-clinical, clinical studies and marketing authorization. This review summarizes the existing regulations/guidelines in US, EU, India, and the associated challenges in developing SCBP with emphasis on clinical aspect.