Table 1. Gene therapy for PID, from SCID... | Rockefeller University Press

Table 1.

Gene therapy for PID, from SCID to other diseases

	Selective advantage	Lifespan of transduced cells	Gene expression profile	Gene expression regulation
SCID	+++	++ (T)	Ubiquitous^a	Looseregulation^b
WAS	+	++ (T), – (platelets)	Ubiquitous	Loose regulation
HLH	–	+	Restricted	Loose regulation
IPEX	–	+	Restricted	Loose regulation
CGD and other myeloid defects	–	–	Restricted	Loose regulation
CD40L deficiency	–	+	Restricted	Tight regulation
XLA (BTK deficiency)	+	+/– (B)	Restricted	Tight regulation^c

	Selective advantage	Lifespan of transduced cells	Gene expression profile	Gene expression regulation
SCID	+++	++ (T)	Ubiquitous^a	Looseregulation^b
WAS	+	++ (T), – (platelets)	Ubiquitous	Loose regulation
HLH	–	+	Restricted	Loose regulation
IPEX	–	+	Restricted	Loose regulation
CGD and other myeloid defects	–	–	Restricted	Loose regulation
CD40L deficiency	–	+	Restricted	Tight regulation
XLA (BTK deficiency)	+	+/– (B)	Restricted	Tight regulation^c

CD40L, CD40 ligand; XLA, X-linked agammaglobulinemia.

Some genes associated with SCID have a restricted expression pattern, i.e., RAG-1, RAG-2, CD3 subunit.

RAG-1, RAG-1 expression is limited in time. Permanent expression might be a problem in gene therapy of SCID caused by RAG-1 or RAG-2 deficiency.

Overexpression of BTK might be harmful.