Genetics | Physician's Weekly


Gene Therapy Effective for Treating Rare Immunodeficiency

Pilot studies report event-free survival following novel gene therapy for ADA-SCID Treatment of young children with adenosine deaminase (ADA) deficiency (ADA-SCID) with an investigational ex vivo lentiviral hematopoietic stem...

Use of SNP Chips to Detect Rare Pathogenic Variants

Single gene disorders that are caused by genetic variants are rare, and finding them can lead to a high probability of disease in an individual and their family. This study aims to determine whether the specificity and...

Kids May Not Be ’Hard-Wired’ for Obesity

Study assessed nature and nurture Genetics can’t necessarily be blamed for excess weight in kids, according to a study from Germany, which suggested that genes played a minor role in weight reduction by lifestyle in...

Givosiran Cuts Porphyria Attacks but Comes At a Price

The RNA interference therapy led to higher frequency of hepatic and renal adverse events Patients with acute intermittent porphyria treated with RNA interference (RNAi) had a significantly lower rate of attacks, and better...



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