Abstract
Duchenne muscular dystrophy (DMD) is a fatal muscle disease caused by the lack of dystrophin, which maintains muscle membrane integrity. We used an adenine base editor (ABE) to modify splice donor sites of the dystrophin gene, causing skipping of a common DMD deletion mutation of exon 51 (∆Ex51) in cardiomyocytes derived from human induced pluripotent stem cells, restoring dystrophin expression. Prime editing was also capable of reframing the dystrophin open reading frame in these cardiomyocytes. Intramuscular injection of ∆Ex51 mice with adeno-associated virus serotype-9 encoding ABE components as a split-intein trans-splicing system allowed gene editing and disease correction in vivo. Our findings demonstrate the effectiveness of nucleotide editing for the correction of diverse DMD mutations with minimal modification of the genome, although improved delivery methods will be required before these strategies can be used to sufficiently edit the genome in patients with D
It s easier than you might think
Written By:
Dr. Jaclyn Lewis Albin / American Academy of Pediatrics | 7:00 am, Apr. 29, 2021 ×
When offering your baby new foods, try to include fruits and vegetables in all the colors of the rainbow. Also offer good sources of protein (such as beans, chicken, fish, and yogurt), fat and iron (for example, iron-fortified oat cereal or meats). Dreamstime / TNS
Q: Is it OK if I make food for my baby at home?
A: Yes. You may find several benefits to feeding your baby homemade foods.
It can be less expensive than the baby food found in grocery stores, for example. It can let your child enjoy baby-friendly versions of foods he or she sees the rest of the family eating, and it may be easier than you think. All you need is a blender, food processor or even a good hand-held mixer.
Written by Jennifer Huizen on April 25, 2021 Fact checked by Rita Ponce, Ph.D.
New research offers promise for a novel form of insulin treatment. Javier Zayas Photography/Getty Images
Two phase 2 trials including hundreds of participants with type 2 diabetes from several countries assessed the safety and efficacy of a long-acting form of insulin called “insulin icodec.”
One trial found that using injectable insulin icodec once a week was as effective as using a standard injectable form of insulin once a day.
The other trial found that it seems to be safe and effective to transition from a standard daily insulin injectable to weekly icodec injections.
Promising Data on Once-Weekly Insulin, Phase 3 Study Ongoing medscape.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from medscape.com Daily Mail and Mail on Sunday newspapers.