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10 Key Learnings from Successful Cellular and Gene Therapy Trials for Rare Diseases

XTalks

These are, in my view, the most valid comparisons, particularly in inherited diseases where the genotype is obviously the same in each individual patient, and as a consequence, the difference in phenotype after gene therapy is a true difference,” he adds. As a result, there are several EU regulations and directives to follow.

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Why a recent advancement is a giant leap for human genomics

Drug Discovery World

The new sequences contain largely highly repetitive DNA structures such as centromeres, telomeres, acrocentric genomic regions, where massive arrays of tandem repeats predominate, and heterochromatin. iv) Highly repetitive regions contain some of the highest mutation rates.

Genome 52
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Biorepositories as a Guiding Resource for Research & Drug Discovery

XTalks

Through these collaborations, cohort research dataset sharing programs allow for whole-genome sequence and genotype data to be made available for independent research. She says dynamics with respect to research goals, priorities and regulations have changed over the past decade. Genotype Data Curation and Return of Results.