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

Human Genetics:

Human genetics is both a basic and connected science. As a key science, it is a piece of hereditary qualities  the part of science that analyses the laws of capacity, transmission, and acknowledgment of data for improvement and capacity of living beings. Inside this system, Human genetics qualities worry about the most fascinating life form – the person. This worry with our very own species influences us to investigate logical outcomes in human hereditary qualities for their hypothetical noteworthiness as well as for their down to earth and incentive for human welfare. Consequently, Human genetics qualities are additionally a connected science. Its incentive for human welfare will undoubtedly have repercussions for hypothetical research too since it impacts the choice of issues by human geneticists, their preparation, and the financing of their exploration. In view of its proceeded with hypothetical and functional intrigue, human hereditary qualities offer interest and human satisfaction unparalleled by work in fields that are either fundamentally hypothetical or completely viable in the subject matter.

Gene Editing

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.

• Genetic Diseases
• Genetic Testing
• Genetic counselling

Stem Cell in Gene Therapy:

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.

• Regenerative therapy for muscular disorders
• CRISPR and Stem cells
• Cord blood stem cell banking
• Cord stem cells for heart failure
• Advances in Hematopoietic stem cell transplantation

Pediatric Geneticist:

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:

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 therapy. 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.

• Process of gene therapy
• Gene therapy products
• Different vectors for gene therapy
• Gene therapy for malignant melanoma
• Gene therapy for sickle-cell disease
• Development of regenerative treatment models

Genetic Disorder:

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:

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.

Nano Therapy

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 on Cell & Gene Therapy:

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:

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.

• Genomics biomarkers
• Digital Biomarkers: Biosensors and Wearable’s
• Immuno-Oncology Biomarkers
• Molecular Biomarkers
• Biomarkers in Clinical Research and Development

Regulatory and Safety Aspects of Cell and Gene Therapy:

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.

Today’s Market Study Genetic Disorders  in USA | Europe | Middle East | Asia Pacific

Asia pacific: The global gene expression analysis market is projected to reach USD 5.30 Billion by 2020 from USD 3.39 Billion in 2015, at a CAGR of 9.3% from 2015 to 2020. The primary growth engine in this region is China, which is expected to register a growth rate of 13.2% in the forecast period.

USA:  The USA Genetics market is poised to reach USD 19.99 Billion by 2020, growing at a CAGR of 9.9% during the forecast period of 2015 to 2020.

Europe:  The Europe genetic Therapy market is estimated at USD 1.22 billion in 2016, and is projected to reach USD 1.8 billion by 2021, growing with a compound annual growth rate (CAGR) of 8.2% during the forecast period, 2016-2021.

Middle East:  The global Human Genetics identification market was valued at an estimated $419.4 million in 2013 and is expected to grow at a CAGR of 13.9% in the next five years.

Market Analysis

The global market for Genetic Disorders is forecast to reach US$2.4 billion by 2018. Increasing knowledge about the potential benefits in genetic Therapy is one of the prime reasons for the growth of the genetic testing market. Advancements in the genetic testing space, aging population and a subsequent rise in the number of chronic diseases, and increasing incidence of cancer cases are the other factors propelling growth in the genetic testing market.

The regional analysis includes North America, Europe, Asia Pacific, Middle East and rest of the world.

The global genetic disorders market expected to grow with a CAGR of more than 10% in the upcoming years. The rise of growth rate is due to increase in technological advancement and various measures taken by US government to expand the national DNA databases.

The Duchene Muscular Dystrophy (DMD) treatment market is expected to change radically by 2019 in the six major pharmaceutical markets

Generic corticosteroids, namely prednisone and deflazacort, remain the current standards of care and contribute to the nascent DMD market, which was estimated at $8.2 million across the 6MM in 2014. However, both the treatment landscape and the DMD market are set to witness tremendous growth—reaching nearly $1 billion by 2019—as a result of the launch of novel mutation-specific drugs in the coming years. However, a large segment of DMD patients are expected to be ineligible for these promising new drugs, leaving vast opportunity for developers to deliver new therapies and for continued growth in the DMD market beyond 2019.

USA: Approximately, more than 24285 members involved in Genetics and related researches.
Europe: Approximately, more than 56083 members involved in Genetics and related researches.
Global: Approximately, 1291100 members involved in Genetics and related researches.

behaviour and decision-making related to genetics and molecular biology, biotechnology, pharmaceuticals, medicals and academia.

Conference on Genetic disorders and Gene Therapy is a much celebrated conference which basically deals with the latest research and developments in the sphere of genetics and molecular biology. This Conference will provide a perfect platform to all the International mix of leading Research Scholars, and Scientists achieved eminence in their field of study, research academicians from the universities and research institutions, industrial research professionals and business associates along with Ph.D. Students to come and inform all the attendees about the latest scientific advancements on the respective sphere