Spencer Knight

𝐂𝐀𝐑 𝐓 𝐓𝐡𝐞𝐫𝐚𝐩𝐲 𝐢𝐧 𝐍𝐨𝐧-𝐎𝐧𝐜𝐨𝐥𝐨𝐠𝐲 👇

CAR T cell therapy has transformed cancer treatment, but its potential extends far beyond oncology.

The figure below illustrates diverse CAR T cell targets in non-oncological diseases:

- Autoimmune diseases,
- Infections,
- Fibrosis,
- Haemophilia,
- Aging.

Exciting see the next-gen CARs tackle areas beyond oncology. Dual-targeting CARs (e.g., CD19-BCMA for lupus) are proving especially effective in tackling treatment-resistant diseases.

#oncology #immunology #celltherapy #CGTweekly

𝐁𝐫𝐞𝐚𝐤𝐭𝐡𝐫𝐨𝐮𝐠𝐡 𝐢𝐧 𝐆𝐞𝐧𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐲:
Sight Restored for Blind Children👇

Amazing news as a gene therapy trial has restored sight in children born with Leber Congenital Amaurosis (LCA), a rare and severe genetic form of blindness.

👉 4 children, aged 1-2, received gene therapy at Moorfields Eye Hospital in London. By injecting healthy copies of the AIPL1 gene into their retinas, doctors have helped them regain the ability to see.

One of these children, Jace, who was legally blind before the treatment and could only distinguish light from dark.

👉 However, after a 60-minute keyhole surgery that introduced the AIPL1 gene into his retina using viral vectors, Jace and the other children can now see shapes, identify toys, recognise their parents' faces, and even read and write.

Amazing. The future of gene therapy is certainly brighter than ever.

Remarkable work and congrats to UCL, Moorfields and MeiraGTx!

#genetherapy #ophthalmology #cellandgenetherapy #CGTweekly