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The One Cell Behind Every Live-cel Treatment — DECODE No. 7
Glafabra
(Last time: gentle outpatient conditioning with melphalan.)
Every Live-cel treatment starts with a rare cell harvested from the patient — one that can rebuild the entire blood system and carry a corrected gene to the body for years. Scientists find it by a marker on its surface: a protein tag written as "CD" followed by a number.
Which cell — and which number? Drop your guess in the ...more
How Did Gene Therapy Patients Go Home the Same Day? — DECODE No. 6
(Last time: lentiviral vectors, and why Live-cel can be re-dosed.)
Older cell-based gene therapies required hospital admission and weeks of recovery — a barrier so heavy many patients and doctors said no. In the FACTS trial, four of five patients did this same step and went home the same day.
What gentler approach made same-day discharge possible? Drop your guess in the comments belo...more
Why Can Some Gene Therapies Be Re-Dosed and Others Can't? — DECODE No. 5
(Last time: the biweekly ERT infusion schedule.)
Some gene therapies can only be given once — the immune system slams the door on a second dose. Live-cel can be given again when it's needed. The difference comes down to what carries the gene into the cell.
What kind of delivery system lets Live-cel be re-dosed? Drop your guess in the comments below.
Follow the whole series: ht...more
How Can Fixing a Few Cells Treat the Whole Body? — DECODE No. 3
(Last time: lyso-Gb3, the biomarker that tracks Fabry disease.)
Here's a beautiful shortcut in biology: to treat the whole body, you don't have to fix every cell. Fix some, and they secrete a helper the neighboring cells absorb and put to work — spreading the benefit outward.
There's a two-word name for this generous phenomenon. What is it? Drop your guess in the comments below.
&nbs...more
